{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,13]],"date-time":"2026-10-13T04:21:03Z","timestamp":1791865263711,"version":"4.3.4"},"reference-count":222,"publisher":"MDPI AG","issue":"19","license":[{"start":{"date-parts":[[2025,9,28]],"date-time":"2025-09-28T00:00:00Z","timestamp":1759017600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["www.mdpi.com"],"crossmark-restriction":true},"short-container-title":["Cells"],"abstract":"<jats:p>Huntington\u2019s Disease (HD) is an inherited neurodegenerative condition caused by an expansion of CAG repeats in the Huntingtin (HTT) gene, leading to a toxic form of the HTT protein. Despite advances in understanding the disease and developing symptomatic treatments, effective therapies for modifying its progression remain limited. Among emerging and novel treatments for central nervous system (CNS) disorders, gene therapy (GT), particularly using adeno-associated virus (AAV)-mediated gene delivery, holds great promise. Numerous preclinical and clinical trials are exploring the benefits of AAVs for treating neurodegenerative and genetic diseases. However, while widely used and investigated in rare and genetic disease treatment, AAVs\u2019 potential for HD treatment remains underexplored. The absence of a comprehensive collection of previous reports, advancements, and methodologies regarding exclusively AAV-mediated GT for HD is notable and prompted us to address this gap. The current review compiles the available and emerging information regarding the application of AAVs in HD therapy, outlines the promise of this approach, and highlights the necessity of conducting further studies to achieve efficient HD treatment. The authors hope that the current review will guide further research to unlock the full potential of AAVs in treating HD.<\/jats:p>","DOI":"10.3390\/cells14191514","type":"journal-article","created":{"date-parts":[[2025,9,29]],"date-time":"2025-09-29T10:49:34Z","timestamp":1759142974000},"page":"1514","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Revolutionizing Huntington\u2019s Disease Treatment: Breakthroughs in AAV-Mediated Gene Therapy"],"prefix":"10.3390","volume":"14","author":[{"given":"Pedram Moeini","family":"Gavgani","sequence":"first","affiliation":[{"name":"DNA & RNA Medicine Division, CIMA-Universidad de Navarra, 31008 Pamplona, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mario","family":"Garc\u00eda-Dom\u00ednguez","sequence":"additional","affiliation":[{"name":"Program of Immunology and Immunotherapy, CIMA-Universidad de Navarra, 31008 Pamplona, Spain"},{"name":"Department of Immunology and Immunotherapy, Cl\u00ednica Universidad de Navarra, 31008 Pamplona, Spain"},{"name":"Centro de Investigaci\u00f3n Biom\u00e9dica en Red de C\u00e1ncer (CIBERONC), 28029 Madrid, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2025,9,28]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"15005","DOI":"10.1038\/nrdp.2015.5","article-title":"Huntington disease","volume":"1","author":"Bates","year":"2015","journal-title":"Nat. Rev. Dis. Primers"},{"key":"ref_2","first-page":"tre-5-296","article-title":"Chorea: A Journey through History","volume":"5","author":"Vale","year":"2015","journal-title":"Tremor Other Hyperkinet. Mov."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1146\/annurev-med-050710-134457","article-title":"Huntington\u2019s disease: Advocacy driving science","volume":"63","author":"Wexler","year":"2012","journal-title":"Annu. Rev. Med."},{"key":"ref_4","first-page":"cer-11-18","article-title":"A Novel Therapy for Huntington\u2019s Disease","volume":"2018","year":"2018","journal-title":"Cerebrum"},{"key":"ref_5","doi-asserted-by":"crossref","unstructured":"Tong, H., Yang, T., Xu, S., Li, X., Liu, L., Zhou, G., Yang, S., Yin, S., Li, X.-J., and Li, S. (2024). Huntington\u2019s Disease: Complex Pathogenesis and Therapeutic Strategies. Int. J. Mol. Sci., 25.","DOI":"10.3390\/ijms25073845"},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"4408","DOI":"10.1093\/brain\/awad353","article-title":"Unravelling the role of huntingtin: From neurodevelopment to neurodegeneration","volume":"146","author":"Schultz","year":"2023","journal-title":"Brain"},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"304","DOI":"10.1016\/j.cels.2022.01.005","article-title":"Dynamics of huntingtin protein interactions in the striatum identifies candidate modifiers of Huntington disease","volume":"13","author":"Greco","year":"2022","journal-title":"Cell Syst."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1385\/MN:28:3:259","article-title":"Wild-type huntingtin plays a role in brain development and neuronal survival","volume":"28","author":"Reiner","year":"2003","journal-title":"Mol. Neurobiol."},{"key":"ref_9","doi-asserted-by":"crossref","unstructured":"Li, X., Tong, H., Xu, S., Zhou, G., Yang, T., Yin, S., Yang, S., Li, X., and Li, S. (2024). Neuroinflammatory Proteins in Huntington\u2019s Disease: Insights into Mechanisms, Diagnosis, and Therapeutic Implications. Int. J. Mol. Sci., 25.","DOI":"10.3390\/ijms252111787"},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"243","DOI":"10.3233\/JHD-220538","article-title":"Mutant Huntingtin Protein Interaction Map Implicates Dysregulation of Multiple Cellular Pathways in Neurodegeneration of Huntington\u2019s Disease","volume":"11","author":"Podvin","year":"2022","journal-title":"J. Huntington\u2019s Dis."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"1107323","DOI":"10.3389\/fmolb.2023.1107323","article-title":"Ubiquitin-modifying enzymes in Huntington\u2019s disease","volume":"10","author":"Sap","year":"2023","journal-title":"Front. Mol. Biosci."},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"e382","DOI":"10.1038\/cddis.2012.121","article-title":"An in vitro perspective on the molecular mechanisms underlying mutant huntingtin protein toxicity","volume":"3","author":"Cisbani","year":"2012","journal-title":"Cell Death Dis."},{"key":"ref_13","doi-asserted-by":"crossref","first-page":"105092","DOI":"10.1016\/j.nbd.2020.105092","article-title":"Inconsistencies in histone acetylation patterns among different HD model systems and HD post-mortem brains","volume":"146","author":"Narayan","year":"2020","journal-title":"Neurobiol. Dis."},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"117827","DOI":"10.1016\/j.biopha.2025.117827","article-title":"The role of mitochondrial dysfunction in Huntington\u2019s disease: Implications for therapeutic targeting","volume":"183","author":"Joshi","year":"2025","journal-title":"Biomed. Pharmacother."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"346","DOI":"10.1111\/jnc.14723","article-title":"Alterations in synaptic function and plasticity in Huntington disease","volume":"150","author":"Sepers","year":"2019","journal-title":"J. Neurochem."},{"key":"ref_16","doi-asserted-by":"crossref","first-page":"e2020293118","DOI":"10.1073\/pnas.2020293118","article-title":"Impaired inhibitory GABAergic synaptic transmission and transcription studied in single neurons by Patch-seq in Huntington\u2019s disease","volume":"118","author":"Paraskevopoulou","year":"2021","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"1175522","DOI":"10.3389\/fnmol.2023.1175522","article-title":"Altered exocytosis of inhibitory synaptic vesicles at single presynaptic terminals of cultured striatal neurons in a knock-in mouse model of Huntington\u2019s disease","volume":"16","author":"Xu","year":"2023","journal-title":"Front. Mol. Neurosci."},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1007\/BF02257483","article-title":"Structure and expression of the Huntington\u2019s disease gene: Evidence against simple inactivation due to an expanded CAG repeat","volume":"20","author":"Ambrose","year":"1994","journal-title":"Somat. Cell Mol. Genet."},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"1541","DOI":"10.1093\/hmg\/2.10.1541","article-title":"Differential 3\u2032 polyadenylation of the Huntington disease gene results in two mRNA species with variable tissue expression","volume":"2","author":"Lin","year":"1993","journal-title":"Hum. Mol. Genet."},{"key":"ref_20","doi-asserted-by":"crossref","unstructured":"Liu, L., Tong, H., Sun, Y., Chen, X., Yang, T., Zhou, G., Li, X.J., and Li, S. (2023). Huntingtin Interacting Proteins and Pathological Implications. Int. J. Mol. Sci., 24.","DOI":"10.3390\/ijms241713060"},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1038\/s41418-021-00914-9","article-title":"The evolutionary history of the polyQ tract in huntingtin sheds light on its functional pro-neural activities","volume":"29","author":"Iennaco","year":"2022","journal-title":"Cell Death Differ."},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"910","DOI":"10.1016\/j.neuron.2016.02.003","article-title":"The Biology of Huntingtin","volume":"89","author":"Saudou","year":"2016","journal-title":"Neuron"},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.expneurol.2011.12.013","article-title":"Protein aggregates in Huntington\u2019s disease","volume":"238","author":"Arrasate","year":"2012","journal-title":"Exp. Neurol."},{"key":"ref_24","first-page":"65","article-title":"The biological function of the Huntingtin protein and its relevance to Huntington\u2019s Disease pathology","volume":"5","author":"Schulte","year":"2011","journal-title":"Curr. Trends Neurol."},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"507","DOI":"10.1111\/jnc.14853","article-title":"The pathobiology of perturbed mutant huntingtin protein-protein interactions in Huntington\u2019s disease","volume":"151","author":"Wanker","year":"2019","journal-title":"J. Neurochem."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"1401","DOI":"10.1056\/NEJM199405193302001","article-title":"A worldwide study of the Huntington\u2019s disease mutation: The sensitivity and specificity of measuring CAG repeats","volume":"330","author":"Kremer","year":"1994","journal-title":"N. Engl. J. Med."},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"314","DOI":"10.1002\/ajmg.b.30970","article-title":"Unstable familial transmissions of Huntington disease alleles with 27-35 CAG repeats (intermediate alleles)","volume":"153B","author":"Semaka","year":"2010","journal-title":"Am. J. Med. Genet. B. Neuropsychiatr. Genet."},{"key":"ref_28","first-page":"16","article-title":"Phenotypic characterization of individuals with 30\u201340 CAG repeats in the Huntington disease (HD) gene reveals HD cases with 36 repeats and apparently normal elderly individuals with 36\u201339 repeats","volume":"59","author":"Rubinsztein","year":"1996","journal-title":"Am. J. Hum. Genet."},{"key":"ref_29","doi-asserted-by":"crossref","first-page":"1137","DOI":"10.1212\/WNL.44.6.1137","article-title":"CAG repeat size and clinical presentation in Huntington\u2019s disease","volume":"44","author":"Ashizawa","year":"1994","journal-title":"Neurology"},{"key":"ref_30","doi-asserted-by":"crossref","first-page":"869","DOI":"10.1007\/s10072-019-04177-8","article-title":"Late-onset Huntington\u2019s disease with 40-42 CAG expansion","volume":"41","author":"Capiluppi","year":"2020","journal-title":"Neurol. Sci."},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"623","DOI":"10.1016\/j.cell.2024.11.038","article-title":"Long somatic DNA-repeat expansion drives neurodegeneration in Huntington\u2019s disease","volume":"188","author":"Handsaker","year":"2025","journal-title":"Cell"},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"948","DOI":"10.1007\/s13311-019-00768-7","article-title":"DNA Damage Repair in Huntington\u2019s Disease and Other Neurodegenerative Diseases","volume":"16","author":"Maiuri","year":"2019","journal-title":"Neurotherapeutics"},{"key":"ref_33","doi-asserted-by":"crossref","first-page":"1647","DOI":"10.4161\/rna.26706","article-title":"Aberrantly spliced HTT, a new player in Huntington\u2019s disease pathogenesis","volume":"10","author":"Gipson","year":"2013","journal-title":"RNA Biol."},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"242","DOI":"10.1038\/s41580-022-00545-z","article-title":"The physiology of alternative splicing","volume":"24","author":"Marasco","year":"2023","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"1869","DOI":"10.1016\/j.jmb.2019.01.034","article-title":"Deregulated Splicing Is a Major Mechanism of RNA-Induced Toxicity in Huntington\u2019s Disease","volume":"431","author":"Schilling","year":"2019","journal-title":"J. Mol. Biol."},{"key":"ref_36","doi-asserted-by":"crossref","unstructured":"Zsindely, N., Nagy, G., Si\u00e1gi, F., Farkas, A., and Bodai, L. (2023). Dysregulated miRNA and mRNA Expression Affect Overlapping Pathways in a Huntington\u2019s Disease Model. Int. J. Mol. Sci., 24.","DOI":"10.3390\/ijms241511942"},{"key":"ref_37","doi-asserted-by":"crossref","first-page":"2009","DOI":"10.1093\/brain\/awab087","article-title":"Huntington\u2019s disease-specific mis-splicing unveils key effector genes and altered splicing factors","volume":"144","author":"Elorza","year":"2021","journal-title":"Brain"},{"key":"ref_38","doi-asserted-by":"crossref","first-page":"104720","DOI":"10.1016\/j.ebiom.2023.104720","article-title":"Widespread dysregulation of mRNA splicing implicates RNA processing in the development and progression of Huntington\u2019s disease","volume":"94","author":"Tano","year":"2023","journal-title":"eBioMedicine"},{"key":"ref_39","doi-asserted-by":"crossref","first-page":"105074","DOI":"10.1016\/j.neuint.2021.105074","article-title":"Epigenetic regulation in Huntington\u2019s disease","volume":"148","author":"Hyeon","year":"2021","journal-title":"Neurochem. Int."},{"key":"ref_40","doi-asserted-by":"crossref","first-page":"4529","DOI":"10.1038\/s41467-020-18255-5","article-title":"DNA methylation study of Huntington\u2019s disease and motor progression in patients and in animal models","volume":"11","author":"Lu","year":"2020","journal-title":"Nat. Commun."},{"key":"ref_41","doi-asserted-by":"crossref","first-page":"330","DOI":"10.1038\/ncpneuro0199","article-title":"Mechanisms of disease: Histone modifications in Huntington\u2019s disease","volume":"2","author":"Cha","year":"2006","journal-title":"Nat. Clin. Pract. Neurol."},{"key":"ref_42","doi-asserted-by":"crossref","first-page":"632","DOI":"10.1016\/S1474-4422(22)00120-X","article-title":"A biological classification of Huntington\u2019s disease: The Integrated Staging System","volume":"21","author":"Tabrizi","year":"2022","journal-title":"Lancet Neurol."},{"key":"ref_43","doi-asserted-by":"crossref","first-page":"21","DOI":"10.5334\/tohm.536","article-title":"Influence of Age of Onset on Huntington\u2019s Disease Phenotype","volume":"10","author":"Kwa","year":"2020","journal-title":"Tremor. Other Hyperkinet. Mov."},{"key":"ref_44","first-page":"e28484","article-title":"Huntington\u2019s Disease: A Clinical Review","volume":"14","author":"Andhale","year":"2022","journal-title":"Cureus"},{"key":"ref_45","doi-asserted-by":"crossref","first-page":"S182","DOI":"10.1016\/0303-8467(92)90064-A","article-title":"The spectrum of motor disorders in Huntington\u2019s disease","volume":"94","author":"Yanagisawa","year":"1992","journal-title":"Clin. Neurol. Neurosurg."},{"key":"ref_46","doi-asserted-by":"crossref","first-page":"752","DOI":"10.1111\/bpa.12432","article-title":"Cognitive dysfunction in Huntington\u2019s disease: Mechanisms and therapeutic strategies beyond BDNF","volume":"26","author":"Saavedra","year":"2016","journal-title":"Brain Pathol."},{"key":"ref_47","doi-asserted-by":"crossref","first-page":"191","DOI":"10.31887\/DCNS.2007.9.2\/arosenblatt","article-title":"Neuropsychiatry of Huntington\u2019s disease","volume":"9","author":"Rosenblatt","year":"2007","journal-title":"Dialogues Clin. Neurosci."},{"key":"ref_48","doi-asserted-by":"crossref","first-page":"RRN1288","DOI":"10.1371\/currents.RRN1288","article-title":"Seven-year clinical follow-up of premanifest carriers of Huntington\u2019s disease","volume":"3","author":"Hart","year":"2011","journal-title":"PLoS Curr."},{"key":"ref_49","doi-asserted-by":"crossref","first-page":"325","DOI":"10.1016\/B978-0-12-381328-2.00014-6","article-title":"Genetics and neuropathology of Huntington\u2019s disease","volume":"98","author":"Reiner","year":"2011","journal-title":"Int. Rev. Neurobiol."},{"key":"ref_50","doi-asserted-by":"crossref","first-page":"101358","DOI":"10.1016\/j.arr.2021.101358","article-title":"Systemic manifestation and contribution of peripheral tissues to Huntington\u2019s disease pathogenesis","volume":"69","author":"Chuang","year":"2021","journal-title":"Ageing Res. Rev."},{"key":"ref_51","doi-asserted-by":"crossref","unstructured":"G\u00f3mez-Jaramillo, L., Cano-Cano, F., Gonz\u00e1lez-Montelongo, M.D.C., Campos-Caro, A., Aguilar-Diosdado, M., and Arroba, A.I. (2022). A New Perspective on Huntington\u2019s Disease: How a Neurological Disorder Influences the Peripheral Tissues. Int. J. Mol. Sci., 23.","DOI":"10.3390\/ijms23116089"},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"2275","DOI":"10.1007\/s00415-020-10334-3","article-title":"The sleep and circadian problems of Huntington\u2019s disease: When, why and their importance","volume":"268","author":"Voysey","year":"2021","journal-title":"J. Neurol."},{"key":"ref_53","doi-asserted-by":"crossref","first-page":"80","DOI":"10.1136\/practneurol-2013-000790","article-title":"Diagnostic genetic testing for Huntington\u2019s disease","volume":"15","author":"Craufurd","year":"2015","journal-title":"Pract. Neurol."},{"key":"ref_54","doi-asserted-by":"crossref","unstructured":"(1996). Unified Huntington\u2019s Disease Rating Scale: Reliability and consistency. Huntington Study Group. Mov. Disord., 11, 136\u2013142.","DOI":"10.1002\/mds.870110204"},{"key":"ref_55","doi-asserted-by":"crossref","first-page":"105488","DOI":"10.1016\/j.parkreldis.2023.105488","article-title":"Neuroimaging biomarkers in Huntington\u2019s disease: Preparing for a new era of therapeutic development","volume":"114","author":"Giehl","year":"2023","journal-title":"Park. Relat. Disord."},{"key":"ref_56","doi-asserted-by":"crossref","first-page":"2483","DOI":"10.1007\/s00415-023-11572-x","article-title":"The updated development of blood-based biomarkers for Huntington\u2019s disease","volume":"270","author":"Zhang","year":"2023","journal-title":"J. Neurol."},{"key":"ref_57","doi-asserted-by":"crossref","first-page":"1156","DOI":"10.1136\/jnnp-2012-304636","article-title":"Prevalence of adult Huntington\u2019s disease in the UK based on diagnoses recorded in general practice records","volume":"84","author":"Evans","year":"2013","journal-title":"J. Neurol. Neurosurg. Psychiatry"},{"key":"ref_58","doi-asserted-by":"crossref","first-page":"361","DOI":"10.1159\/000516767","article-title":"Modeling Manifest Huntington\u2019s Disease Prevalence Using Diagnosed Incidence and Survival Time","volume":"55","author":"Crowell","year":"2021","journal-title":"Neuroepidemiology"},{"key":"ref_59","doi-asserted-by":"crossref","first-page":"318","DOI":"10.1186\/s12883-019-1556-3","article-title":"Epidemiology and health care utilization of patients suffering from Huntington\u2019s disease in Germany: Real world evidence based on German claims data","volume":"19","author":"Ohlmeier","year":"2019","journal-title":"BMC Neurol."},{"key":"ref_60","doi-asserted-by":"crossref","first-page":"367","DOI":"10.1111\/cge.12574","article-title":"Epidemiology of Huntington disease: First post-HTT gene analysis of prevalence in Italy","volume":"89","author":"Squitieri","year":"2016","journal-title":"Clin. Genet."},{"key":"ref_61","doi-asserted-by":"crossref","first-page":"212","DOI":"10.1080\/13696998.2022.2033493","article-title":"Epidemiology and economic burden of Huntington\u2019s disease: A Canadian provincial public health system perspective","volume":"25","author":"Shaw","year":"2022","journal-title":"J. Med. Econ."},{"key":"ref_62","first-page":"900","article-title":"Population genetic study of Huntington\u2019s disease\u2014prevalence and founder\u2019s effect in the San-in area, western Japan","volume":"57","author":"Adachi","year":"1999","journal-title":"Nihon Rinsho. Jpn. J. Clin. Med."},{"key":"ref_63","first-page":"43","article-title":"Huntington\u2019s disease in Hong Kong Chinese: Epidemiology and clinical picture","volume":"31","author":"Chang","year":"1994","journal-title":"Clin. Exp. Neurol."},{"key":"ref_64","doi-asserted-by":"crossref","first-page":"2327","DOI":"10.1002\/mds.29228","article-title":"Prevalence and Incidence of Huntington\u2019s Disease: An Updated Systematic Review and Meta-Analysis","volume":"37","author":"Medina","year":"2022","journal-title":"Mov. Disord."},{"key":"ref_65","doi-asserted-by":"crossref","first-page":"963457","DOI":"10.3389\/fpsyt.2022.963457","article-title":"Social cognition and quality of life in Huntington\u2019s disease","volume":"13","author":"Eddy","year":"2022","journal-title":"Front. Psychiatry"},{"key":"ref_66","doi-asserted-by":"crossref","first-page":"124","DOI":"10.1186\/s13023-015-0324-8","article-title":"Unmet needs for healthcare and social support services in patients with Huntington\u2019s disease: A cross-sectional population-based study","volume":"10","author":"Howe","year":"2015","journal-title":"Orphanet J. Rare Dis."},{"key":"ref_67","doi-asserted-by":"crossref","first-page":"11795735221092517","DOI":"10.1177\/11795735221092517","article-title":"Current and Possible Future Therapeutic Options for Huntington\u2019s Disease","volume":"14","author":"Ferguson","year":"2022","journal-title":"J. Cent. Nerv. Syst. Dis."},{"key":"ref_68","doi-asserted-by":"crossref","first-page":"RRN1260","DOI":"10.1371\/currents.RRN1260","article-title":"An International Survey-based Algorithm for the Pharmacologic Treatment of Chorea in Huntington\u2019s Disease","volume":"3","author":"Burgunder","year":"2011","journal-title":"PLoS Curr."},{"key":"ref_69","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1111\/ene.13413","article-title":"Huntington\u2019s disease: A clinical review","volume":"25","author":"McColgan","year":"2018","journal-title":"Eur. J. Neurol."},{"key":"ref_70","doi-asserted-by":"crossref","first-page":"582","DOI":"10.1002\/mdc3.12483","article-title":"Tetrabenazine Versus Deutetrabenazine for Huntington\u2019s Disease: Twins or Distant Cousins?","volume":"4","author":"Rodrigues","year":"2017","journal-title":"Mov. Disord. Clin. Pract."},{"key":"ref_71","doi-asserted-by":"crossref","first-page":"657","DOI":"10.2147\/NDT.S6430","article-title":"Tetrabenazine: The first approved drug for the treatment of chorea in US patients with Huntington disease","volume":"6","author":"Frank","year":"2010","journal-title":"Neuropsychiatr. Dis. Treat."},{"key":"ref_72","doi-asserted-by":"crossref","first-page":"230","DOI":"10.1097\/01.wnf.0000136892.24629.96","article-title":"Tetrabenazine treatment in movement disorders","volume":"27","author":"Paleacu","year":"2004","journal-title":"Clin. Neuropharmacol."},{"key":"ref_73","doi-asserted-by":"crossref","first-page":"358","DOI":"10.1212\/WNL.48.2.358","article-title":"Long-term effects of tetrabenazine in hyperkinetic movement disorders","volume":"48","author":"Jankovic","year":"1997","journal-title":"Neurology"},{"key":"ref_74","doi-asserted-by":"crossref","first-page":"31","DOI":"10.2217\/nmt-2018-0040","article-title":"Deutetrabenazine in the treatment of Huntington\u2019s disease","volume":"9","author":"Richard","year":"2019","journal-title":"Neurodegener. Dis. Manag."},{"key":"ref_75","doi-asserted-by":"crossref","first-page":"235","DOI":"10.1016\/B978-0-444-63425-2.00010-6","article-title":"The role of dopamine in Huntington\u2019s disease","volume":"211","author":"Cepeda","year":"2014","journal-title":"Prog. Brain Res."},{"key":"ref_76","first-page":"A99","article-title":"Subcutaneous haloperidol for chorea in patients with Huntington\u2019s disease and dysphagia","volume":"93","author":"Lee","year":"2022","journal-title":"J. Neurol. Neurosurg. Psychiatry"},{"key":"ref_77","doi-asserted-by":"crossref","first-page":"110772","DOI":"10.1016\/j.biopha.2020.110772","article-title":"Haloperidol in palliative care: Indications and risks","volume":"132","author":"Oduah","year":"2020","journal-title":"Biomed. Pharmacother."},{"key":"ref_78","doi-asserted-by":"crossref","unstructured":"Feleus, S., van Schaijk, M., Roos, R.A.C., and de Bot, S.T. (2022). The Many Faces of Huntington\u2019s Chorea Treatment: The Impact of Sudden Withdrawal of Tiapride after 40 Years of Use and a Systematic Review. J. Pers. Med., 12.","DOI":"10.3390\/jpm12040589"},{"key":"ref_79","doi-asserted-by":"crossref","first-page":"281","DOI":"10.2165\/00003495-199753020-00007","article-title":"Olanzapine. A review of its pharmacological properties and therapeutic efficacy in the management of schizophrenia and related psychoses","volume":"53","author":"Fulton","year":"1997","journal-title":"Drugs"},{"key":"ref_80","doi-asserted-by":"crossref","first-page":"441","DOI":"10.1034\/j.1600-0404.2002.01197.x","article-title":"Olanzapine in Huntington\u2019s disease","volume":"105","author":"Paleacu","year":"2002","journal-title":"Acta Neurol. Scand."},{"key":"ref_81","doi-asserted-by":"crossref","first-page":"1719","DOI":"10.1176\/appi.ajp.158.10.1719","article-title":"Association of olanzapine-induced weight gain with an increase in body fat","volume":"158","author":"Eder","year":"2001","journal-title":"Am. J. Psychiatry"},{"key":"ref_82","doi-asserted-by":"crossref","first-page":"193","DOI":"10.1358\/dot.1999.35.3.533849","article-title":"Quetiapine fumarate (Seroquel): A new atypical antipsychotic","volume":"35","author":"Goldstein","year":"1999","journal-title":"Drugs Today"},{"key":"ref_83","doi-asserted-by":"crossref","first-page":"1114","DOI":"10.1007\/s00415-002-0741-1","article-title":"Quetiapine in Huntington\u2019s disease: A first case report","volume":"249","author":"Bonelli","year":"2002","journal-title":"J. Neurol."},{"key":"ref_84","doi-asserted-by":"crossref","first-page":"70","DOI":"10.1176\/appi.psy.47.1.70","article-title":"Quetiapine in the treatment of behavioral disturbances in patients with Huntington\u2019s disease","volume":"47","author":"Alpay","year":"2006","journal-title":"Psychosomatics"},{"key":"ref_85","doi-asserted-by":"crossref","first-page":"803","DOI":"10.1517\/14656566.6.5.803","article-title":"Risperidone: A review","volume":"6","year":"2005","journal-title":"Expert Opin. Pharmacother."},{"key":"ref_86","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1002\/mdc3.12706","article-title":"Comparing Risperidone and Olanzapine to Tetrabenazine for the Management of Chorea in Huntington Disease: An Analysis from the Enroll-HD Database","volume":"6","author":"Schultz","year":"2018","journal-title":"Mov. Disord. Clin. Pract."},{"key":"ref_87","first-page":"20","article-title":"Risperidone side effects","volume":"61","author":"Conley","year":"2000","journal-title":"J. Clin. Psychiatry"},{"key":"ref_88","first-page":"1","article-title":"Aripiprazole in the treatment of Huntington\u2019s disease: A case series","volume":"5","author":"Ciammola","year":"2009","journal-title":"Neuropsychiatr. Dis. Treat."},{"key":"ref_89","doi-asserted-by":"crossref","first-page":"111023","DOI":"10.1016\/j.jpsychores.2022.111023","article-title":"The effects of antidepressants on depressive symptoms in manifest Huntington\u2019s disease","volume":"162","author":"Ogilvie","year":"2022","journal-title":"J. Psychosom. Res."},{"key":"ref_90","doi-asserted-by":"crossref","first-page":"387","DOI":"10.3390\/neurolint13030038","article-title":"Selective Serotonin Reuptake Inhibitors and Adverse Effects: A Narrative Review","volume":"13","author":"Edinoff","year":"2021","journal-title":"Neurol. Int."},{"key":"ref_91","doi-asserted-by":"crossref","first-page":"401","DOI":"10.1002\/mds.25750","article-title":"Results of the citalopram to enhance cognition in Huntington disease trial","volume":"29","author":"Beglinger","year":"2014","journal-title":"Mov. Disord."},{"key":"ref_92","first-page":"20","article-title":"Long-term side effects of SSRIs: Sexual dysfunction and weight gain","volume":"64","author":"Hirschfeld","year":"2003","journal-title":"J. Clin. Psychiatry"},{"key":"ref_93","doi-asserted-by":"crossref","first-page":"1356","DOI":"10.1176\/appi.ajp.2007.07030454","article-title":"Early evidence on the effects of regulators\u2019 suicidality warnings on SSRI prescriptions and suicide in children and adolescents","volume":"164","author":"Gibbons","year":"2007","journal-title":"Am. J. Psychiatry"},{"key":"ref_94","doi-asserted-by":"crossref","first-page":"11","DOI":"10.5214\/ans.0972.7531.220104","article-title":"Neuroprotective potential of escitalopram against behavioral, mitochondrial and oxidative dysfunction induced by 3-nitropropionic acid","volume":"22","author":"Shetty","year":"2015","journal-title":"Ann. Neurosci."},{"key":"ref_95","doi-asserted-by":"crossref","first-page":"264","DOI":"10.1007\/s002130000575","article-title":"Fluoxetine in the treatment of Huntington\u2019s disease","volume":"153","author":"Daniele","year":"2001","journal-title":"Psychopharmacology"},{"key":"ref_96","doi-asserted-by":"crossref","first-page":"481","DOI":"10.2165\/00003495-198632060-00002","article-title":"Fluoxetine. A review of its pharmacodynamic and pharmacokinetic properties, and therapeutic efficacy in depressive illness","volume":"32","author":"Benfield","year":"1986","journal-title":"Drugs"},{"key":"ref_97","doi-asserted-by":"crossref","first-page":"338","DOI":"10.1176\/jnp.8.3.338","article-title":"Sertraline in the treatment of severe aggressiveness in Huntington\u2019s disease","volume":"8","author":"Ranen","year":"1996","journal-title":"J. Neuropsychiatry Clin. Neurosci."},{"key":"ref_98","first-page":"1813","article-title":"Pictorial review of glutamate excitotoxicity: Fundamental concepts for neuroimaging","volume":"22","author":"Mark","year":"2001","journal-title":"Am. J. Neuroradiol."},{"key":"ref_99","doi-asserted-by":"crossref","first-page":"694","DOI":"10.1212\/WNL.59.5.694","article-title":"Huntington\u2019s disease: A randomized, controlled trial using the NMDA-antagonist amantadine","volume":"59","author":"Morris","year":"2002","journal-title":"Neurology"},{"key":"ref_100","doi-asserted-by":"crossref","first-page":"1995","DOI":"10.1212\/01.WNL.0000068165.07883.64","article-title":"IV amantadine improves chorea in Huntington\u2019s disease: An acute randomized, controlled study","volume":"60","author":"Lucetti","year":"2003","journal-title":"Neurology"},{"key":"ref_101","doi-asserted-by":"crossref","first-page":"1343","DOI":"10.1038\/s41598-024-51904-z","article-title":"A randomized open-label clinical trial on the effect of Amantadine on post Covid-19 fatigue","volume":"14","author":"Harandi","year":"2024","journal-title":"Sci. Rep."},{"key":"ref_102","doi-asserted-by":"crossref","unstructured":"Huntington Study Group (2003). Dosage effects of riluzole in Huntington\u2019s disease: A multicenter placebo-controlled study. Neurology, 61, 1551\u20131556.","DOI":"10.1212\/01.WNL.0000096019.71649.2B"},{"key":"ref_103","doi-asserted-by":"crossref","first-page":"262","DOI":"10.1002\/ana.21181","article-title":"Riluzole in Huntington\u2019s disease: A 3-year, randomized controlled study","volume":"62","author":"Landwehrmeyer","year":"2007","journal-title":"Ann. Neurol."},{"key":"ref_104","doi-asserted-by":"crossref","first-page":"738","DOI":"10.4088\/JCP.v65n0601","article-title":"Anticonvulsants as mood stabilizers and adjuncts to antipsychotics: Valproate, lamotrigine, carbamazepine, and oxcarbazepine and actions at voltage-gated sodium channels","volume":"65","author":"Stahl","year":"2004","journal-title":"J. Clin. Psychiatry"},{"key":"ref_105","doi-asserted-by":"crossref","first-page":"14","DOI":"10.1186\/1745-0179-5-14","article-title":"Anticonvulsants in the treatment of aggression in the demented elderly: An update","volume":"5","author":"Amann","year":"2009","journal-title":"Clin. Pract. Epidemiol. Ment. Health"},{"key":"ref_106","doi-asserted-by":"crossref","first-page":"723","DOI":"10.1111\/pcn.12186","article-title":"Potential role of anticonvulsants in the treatment of obsessive-compulsive and related disorders","volume":"68","author":"Wang","year":"2014","journal-title":"Psychiatry Clin. Neurosci."},{"key":"ref_107","doi-asserted-by":"crossref","first-page":"589","DOI":"10.1007\/s11096-018-0641-9","article-title":"Comparative efficacy of antiepileptic drugs for patients with generalized epileptic seizures: Systematic review and network meta-analyses","volume":"40","author":"Campos","year":"2018","journal-title":"Int. J. Clin. Pharm."},{"key":"ref_108","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1016\/S0074-7742(06)81006-8","article-title":"Mechanisms of action of antiepileptic drugs","volume":"81","author":"White","year":"2007","journal-title":"Int. Rev. Neurobiol."},{"key":"ref_109","doi-asserted-by":"crossref","first-page":"2050","DOI":"10.1038\/sj.npp.1300245","article-title":"Valproate and GABAergic system effects","volume":"28","author":"Winterer","year":"2003","journal-title":"Neuropsychopharmacology"},{"key":"ref_110","doi-asserted-by":"crossref","first-page":"973","DOI":"10.1056\/NEJM200009283431316","article-title":"Improvement of Huntington\u2019s disease with olanzapine and valproate","volume":"343","author":"Grove","year":"2000","journal-title":"N. Engl. J. Med."},{"key":"ref_111","doi-asserted-by":"crossref","first-page":"e1","DOI":"10.4102\/phcfm.v12i1.2162","article-title":"General side effects and challenges associated with anti-epilepsy medication: A review of related literature","volume":"12","author":"Mutanana","year":"2020","journal-title":"Afr. J. Prim. Health Care Fam. Med."},{"key":"ref_112","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1023\/A:1014814924965","article-title":"Mechanisms of action of carbamazepine and its derivatives, oxcarbazepine, BIA 2-093, and BIA 2-024","volume":"27","author":"Carvalho","year":"2002","journal-title":"Neurochem. Res."},{"key":"ref_113","doi-asserted-by":"crossref","first-page":"256","DOI":"10.1002\/ccr3.3509","article-title":"Carbamazepine-induced Stevens-Johnson syndrome in a patient with history of methotrexate-induced mast cell activation syndrome","volume":"9","author":"Zaalouk","year":"2020","journal-title":"Clin. Case Rep."},{"key":"ref_114","doi-asserted-by":"crossref","first-page":"123","DOI":"10.4103\/0972-5229.83018","article-title":"Carbamzepine-induced toxic epidermal necrolysis","volume":"15","author":"Chowta","year":"2011","journal-title":"Indian J. Crit. Care Med."},{"key":"ref_115","doi-asserted-by":"crossref","first-page":"190","DOI":"10.1016\/0006-8993(93)91660-K","article-title":"Studies on the mechanism of action of the novel anticonvulsant lamotrigine (Lamictal) using primary neurological cultures from rat cortex","volume":"612","author":"Lees","year":"1993","journal-title":"Brain Res."},{"key":"ref_116","doi-asserted-by":"crossref","first-page":"219","DOI":"10.1016\/j.neulet.2006.08.036","article-title":"Evaluation of clinically relevant glutamate pathway inhibitors in in vitro model of Huntington\u2019s disease","volume":"407","author":"Wu","year":"2006","journal-title":"Neurosci. Lett."},{"key":"ref_117","doi-asserted-by":"crossref","first-page":"645","DOI":"10.1016\/j.jpainsymman.2018.07.012","article-title":"Levetiracetam","volume":"56","author":"Howard","year":"2018","journal-title":"J. Pain Symptom Manag."},{"key":"ref_118","doi-asserted-by":"crossref","first-page":"280","DOI":"10.1097\/01.wnf.0000186648.93612.a5","article-title":"Efficacy of levetiracetam in Huntington disease","volume":"28","author":"Sciruicchio","year":"2005","journal-title":"Clin. Neuropharmacol."},{"key":"ref_119","doi-asserted-by":"crossref","first-page":"1722","DOI":"10.1016\/j.brs.2020.09.025","article-title":"An individual patient analysis of the efficacy of using GPi-DBS to treat Huntington\u2019s disease","volume":"13","author":"Yin","year":"2020","journal-title":"Brain Stimul."},{"key":"ref_120","doi-asserted-by":"crossref","first-page":"1258190","DOI":"10.3389\/fnagi.2023.1258190","article-title":"Deep brain stimulation in PD: Risk of complications, morbidity, and hospitalizations: A systematic review","volume":"15","author":"Olson","year":"2023","journal-title":"Front. Aging Neurosci."},{"key":"ref_121","doi-asserted-by":"crossref","first-page":"820","DOI":"10.2522\/ptj.20070346","article-title":"Physical therapy intervention for people with Huntington disease","volume":"88","author":"Busse","year":"2008","journal-title":"Phys. Ther."},{"key":"ref_122","doi-asserted-by":"crossref","first-page":"e28","DOI":"10.12786\/bn.2023.16.e28","article-title":"Update of Rehabilitation in Huntington\u2019s Disease: Narrative Review","volume":"16","author":"Kim","year":"2023","journal-title":"Brain Neurorehabil."},{"key":"ref_123","first-page":"1","article-title":"A home program of speech therapy in Huntington\u2019s disease","volume":"18","author":"Giddens","year":"2010","journal-title":"J. Med. Speech Lang. Pathol."},{"key":"ref_124","doi-asserted-by":"crossref","first-page":"485","DOI":"10.3233\/JHD-210488","article-title":"Quantifying the Onset of Unintended Weight Loss in Huntington\u2019s Disease: A Retrospective Analysis of Enroll-HD","volume":"10","author":"Ogilvie","year":"2021","journal-title":"J. Huntington\u2019s Dis."},{"key":"ref_125","doi-asserted-by":"crossref","first-page":"231","DOI":"10.3233\/JHD-200418","article-title":"Psychological Interventions for People with Huntington\u2019s Disease: A Call to Arms","volume":"9","author":"Zarotti","year":"2020","journal-title":"J. Huntington\u2019s Dis."},{"key":"ref_126","doi-asserted-by":"crossref","first-page":"71","DOI":"10.3233\/JHD-230565","article-title":"Supporting Huntington\u2019s Disease Families Through the Ups and Downs of Clinical Trials","volume":"12","author":"Andrew","year":"2023","journal-title":"J. Huntington\u2019s Dis."},{"key":"ref_127","doi-asserted-by":"crossref","first-page":"585","DOI":"10.1177\/0022034509337480","article-title":"Gene therapy: Design and prospects for craniofacial regeneration","volume":"88","author":"Scheller","year":"2009","journal-title":"J. Dent. Res."},{"key":"ref_128","doi-asserted-by":"crossref","unstructured":"Li, X., Le, Y., Zhang, Z., Nian, X., Liu, B., and Yang, X. (2023). Viral Vector-Based Gene Therapy. Int. J. Mol. Sci., 24.","DOI":"10.3390\/ijms24097736"},{"key":"ref_129","doi-asserted-by":"crossref","unstructured":"Wang, C., Pan, C., Yong, H., Wang, F., Bo, T., Zhao, Y., Ma, B., He, W., and Li, M. (2023). Emerging non-viral vectors for gene delivery. J. Nanobiotechnology, 21.","DOI":"10.1186\/s12951-023-02044-5"},{"key":"ref_130","doi-asserted-by":"crossref","first-page":"e67","DOI":"10.1002\/cpns.67","article-title":"Recombinant Viral Vectors as Neuroscience Tools","volume":"87","author":"Chen","year":"2019","journal-title":"Curr. Protoc. Neurosci."},{"key":"ref_131","doi-asserted-by":"crossref","first-page":"86","DOI":"10.2174\/1566523214666140302193709","article-title":"Basic biology of adeno-associated virus (AAV) vectors used in gene therapy","volume":"14","author":"Balakrishnan","year":"2014","journal-title":"Curr. Gene Ther."},{"key":"ref_132","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1146\/annurev-virology-031413-085355","article-title":"AAV-Mediated Gene Therapy for Research and Therapeutic Purposes","volume":"1","author":"Samulski","year":"2014","journal-title":"Annu. Rev. Virol."},{"key":"ref_133","doi-asserted-by":"crossref","first-page":"2898","DOI":"10.1016\/j.ymthe.2021.08.031","article-title":"AAV integration in human hepatocytes","volume":"29","author":"Dalwadi","year":"2021","journal-title":"Mol. Ther."},{"key":"ref_134","doi-asserted-by":"crossref","first-page":"1177","DOI":"10.1038\/mt.2012.47","article-title":"Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver","volume":"20","author":"Nowrouzi","year":"2012","journal-title":"Mol. Ther."},{"key":"ref_135","doi-asserted-by":"crossref","first-page":"709","DOI":"10.1016\/j.ymthe.2020.01.001","article-title":"Immune Responses to Viral Gene Therapy Vectors","volume":"28","author":"Shirley","year":"2020","journal-title":"Mol. Ther."},{"key":"ref_136","doi-asserted-by":"crossref","first-page":"836","DOI":"10.1089\/hum.2023.119","article-title":"Understanding and Tackling Immune Responses to Adeno-Associated Viral Vectors","volume":"34","author":"Unzu","year":"2023","journal-title":"Hum. Gene Ther."},{"key":"ref_137","doi-asserted-by":"crossref","first-page":"1604","DOI":"10.1038\/sj.gt.3300782","article-title":"Inducible long-term gene expression in brain with adeno-associated virus gene transfer","volume":"5","author":"Haberman","year":"1998","journal-title":"Gene Ther."},{"key":"ref_138","doi-asserted-by":"crossref","first-page":"1364","DOI":"10.1016\/j.ymthe.2022.03.004","article-title":"Durability of transgene expression after rAAV gene therapy","volume":"30","author":"Muhuri","year":"2022","journal-title":"Mol. Ther."},{"key":"ref_139","doi-asserted-by":"crossref","first-page":"127","DOI":"10.4103\/ijabmr.IJABMR_190_19","article-title":"Onasemnogene Abeparvovec for Spinal Muscular Atrophy: The Costlier Drug Ever","volume":"9","author":"Mahajan","year":"2019","journal-title":"Int. J. Appl. Basic Med. Res."},{"key":"ref_140","doi-asserted-by":"crossref","first-page":"2073","DOI":"10.1016\/j.ymthe.2020.06.001","article-title":"Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B","volume":"28","author":"George","year":"2020","journal-title":"Mol. Ther."},{"key":"ref_141","doi-asserted-by":"crossref","first-page":"923","DOI":"10.1182\/blood.2019003777","article-title":"Gene therapy for hemophilia: A review on clinical benefit, limitations, and remaining issues","volume":"138","author":"Leebeek","year":"2021","journal-title":"Blood"},{"key":"ref_142","doi-asserted-by":"crossref","first-page":"100759","DOI":"10.1016\/j.blre.2020.100759","article-title":"Discussing investigational AAV gene therapy with hemophilia patients: A guide","volume":"47","author":"Sidonio","year":"2021","journal-title":"Blood Rev."},{"key":"ref_143","doi-asserted-by":"crossref","first-page":"1323","DOI":"10.1007\/s40265-023-01929-x","article-title":"Delandistrogene Moxeparvovec: First Approval","volume":"83","author":"Hoy","year":"2023","journal-title":"Drugs"},{"key":"ref_144","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1089\/hum.2016.087","article-title":"Adeno-Associated Virus-Based Gene Therapy for CNS Diseases","volume":"27","author":"Hocquemiller","year":"2016","journal-title":"Hum. Gene Ther."},{"key":"ref_145","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1016\/j.neuropharm.2016.02.013","article-title":"Viral vectors for therapy of neurologic diseases","volume":"120","author":"Choudhury","year":"2017","journal-title":"Neuropharmacology"},{"key":"ref_146","doi-asserted-by":"crossref","first-page":"839","DOI":"10.1016\/j.neuron.2019.02.017","article-title":"Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality","volume":"101","author":"Hudry","year":"2019","journal-title":"Neuron"},{"key":"ref_147","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1016\/j.jconrel.2022.10.018","article-title":"Nucleic acid therapies for CNS diseases: Pathophysiology, targets, barriers, and delivery strategies","volume":"352","author":"Padmakumar","year":"2022","journal-title":"J. Control. Release"},{"key":"ref_148","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1038\/s41434-022-00346-1","article-title":"Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes","volume":"30","author":"Chandran","year":"2023","journal-title":"Gene Ther."},{"key":"ref_149","doi-asserted-by":"crossref","unstructured":"Daci, R., and Flotte, T.R. (2024). Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders. Int. J. Mol. Sci., 25.","DOI":"10.3390\/ijms25021050"},{"key":"ref_150","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1146\/annurev-neuro-111020-100834","article-title":"Adeno-Associated Virus Toolkit to Target Diverse Brain Cells","volume":"45","author":"Challis","year":"2022","journal-title":"Annu. Rev. Neurosci."},{"key":"ref_151","doi-asserted-by":"crossref","first-page":"254","DOI":"10.1016\/j.omtm.2023.03.013","article-title":"Semi-automated workflows to quantify AAV transduction in various brain areas and predict gene editing outcome for neurological disorders","volume":"29","author":"Duarte","year":"2023","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"ref_152","doi-asserted-by":"crossref","unstructured":"Owusu-Yaw, B.S., Zhang, Y., Garrett, L., Yao, A., Shing, K., Batista, A.R., Sena-Esteves, M., Upadhyay, J., Kegel-Gleason, K., and Todd, N. (2024). Focused Ultrasound-Mediated Disruption of the Blood-Brain Barrier for AAV9 Delivery in a Mouse Model of Huntington\u2019s Disease. Pharmaceutics, 16.","DOI":"10.3390\/pharmaceutics16060710"},{"key":"ref_153","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1038\/nbt.1515","article-title":"Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes","volume":"27","author":"Foust","year":"2009","journal-title":"Nat. Biotechnol."},{"key":"ref_154","doi-asserted-by":"crossref","first-page":"757","DOI":"10.1517\/14712598.2012.681463","article-title":"The advent of AAV9 expands applications for brain and spinal cord gene delivery","volume":"12","author":"Dayton","year":"2012","journal-title":"Expert Opin. Biol. Ther."},{"key":"ref_155","doi-asserted-by":"crossref","first-page":"204","DOI":"10.1038\/nbt.3440","article-title":"Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain","volume":"34","author":"Deverman","year":"2016","journal-title":"Nat. Biotechnol."},{"key":"ref_156","doi-asserted-by":"crossref","first-page":"458","DOI":"10.1016\/j.jconrel.2023.01.067","article-title":"AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges","volume":"355","author":"Kang","year":"2023","journal-title":"J. Control. Release"},{"key":"ref_157","doi-asserted-by":"crossref","first-page":"789","DOI":"10.1038\/s41573-023-00766-7","article-title":"AAV-based in vivo gene therapy for neurological disorders","volume":"22","author":"Ling","year":"2023","journal-title":"Nat. Rev. Drug Discov."},{"key":"ref_158","doi-asserted-by":"crossref","first-page":"1121","DOI":"10.1080\/14712598.2020.1792880","article-title":"AAV5-miHTT gene therapy for Huntington disease: Lowering both huntingtins","volume":"20","author":"Evers","year":"2020","journal-title":"Expert Opin. Biol. Ther."},{"key":"ref_159","doi-asserted-by":"crossref","first-page":"618","DOI":"10.1016\/j.ymthe.2005.05.006","article-title":"Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6\/1 Huntington\u2019s disease transgenic mice","volume":"12","author":"Nash","year":"2005","journal-title":"Mol. Ther."},{"key":"ref_160","doi-asserted-by":"crossref","first-page":"5820","DOI":"10.1073\/pnas.0501507102","article-title":"RNA interference improves motor and neuropathological abnormalities in a Huntington\u2019s disease mouse model","volume":"102","author":"Harper","year":"2005","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_161","doi-asserted-by":"crossref","first-page":"190","DOI":"10.1016\/j.bbrc.2006.02.141","article-title":"rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse","volume":"343","author":"Machida","year":"2006","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"ref_162","doi-asserted-by":"crossref","first-page":"17204","DOI":"10.1073\/pnas.0708285104","article-title":"Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits","volume":"104","author":"DiFiglia","year":"2007","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_163","doi-asserted-by":"crossref","first-page":"947","DOI":"10.1038\/mt.2008.50","article-title":"AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington\u2019s disease","volume":"16","author":"Franich","year":"2008","journal-title":"Mol. Ther."},{"key":"ref_164","doi-asserted-by":"crossref","first-page":"1053","DOI":"10.1038\/mt.2009.17","article-title":"Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington\u2019s disease mice","volume":"17","author":"Boudreau","year":"2009","journal-title":"Mol. Ther."},{"key":"ref_165","doi-asserted-by":"crossref","first-page":"2152","DOI":"10.1038\/mt.2011.219","article-title":"Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington\u2019s disease","volume":"19","author":"McBride","year":"2011","journal-title":"Mol. Ther."},{"key":"ref_166","doi-asserted-by":"crossref","first-page":"275","DOI":"10.1016\/j.omtm.2019.09.010","article-title":"AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes","volume":"15","author":"Keskin","year":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"ref_167","first-page":"E3136","article-title":"Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6\/2 mice","volume":"109","author":"Herrmann","year":"2012","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_168","doi-asserted-by":"crossref","first-page":"1197","DOI":"10.1093\/brain\/awr333","article-title":"Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum","volume":"135","author":"Grondin","year":"2012","journal-title":"Brain"},{"key":"ref_169","doi-asserted-by":"crossref","first-page":"797","DOI":"10.1038\/mt.2013.289","article-title":"Intrajugular vein delivery of AAV9-RNAi prevents neuropathological changes and weight loss in Huntington\u2019s disease mice","volume":"22","author":"Dufour","year":"2014","journal-title":"Mol. Ther."},{"key":"ref_170","doi-asserted-by":"crossref","first-page":"461","DOI":"10.1089\/hum.2013.200","article-title":"Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington\u2019s disease","volume":"25","author":"Stanek","year":"2014","journal-title":"Hum. Gene Ther."},{"key":"ref_171","doi-asserted-by":"crossref","first-page":"1131","DOI":"10.1038\/s41591-019-0478-3","article-title":"Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington\u2019s disease","volume":"25","author":"Zeitler","year":"2019","journal-title":"Nat. Med."},{"key":"ref_172","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1186\/s13024-016-0128-x","article-title":"Deimmunization for gene therapy: Host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice","volume":"11","author":"Mielcarek","year":"2016","journal-title":"Mol. Neurodegener."},{"key":"ref_173","doi-asserted-by":"crossref","first-page":"16037","DOI":"10.1038\/mtm.2016.37","article-title":"Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: Implications for Huntington\u2019s disease","volume":"3","author":"Hadaczek","year":"2016","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"ref_174","doi-asserted-by":"crossref","first-page":"e297","DOI":"10.1038\/mtna.2016.7","article-title":"Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington\u2019s Disease","volume":"5","author":"Miniarikova","year":"2016","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_175","first-page":"7120753","article-title":"An Intrabody Drug (rAAV6-INT41) Reduces the Binding of N-Terminal Huntingtin Fragment(s) to DNA to Basal Levels in PC12 Cells and Delays Cognitive Loss in the R6\/2 Animal Model","volume":"2016","author":"Amaro","year":"2016","journal-title":"J. Neurodegener. Dis."},{"key":"ref_176","doi-asserted-by":"crossref","first-page":"104569","DOI":"10.1016\/j.nbd.2019.104569","article-title":"Antibody-based therapies for Huntington\u2019s disease: Current status and future directions","volume":"132","author":"Denis","year":"2019","journal-title":"Neurobiol. Dis."},{"key":"ref_177","doi-asserted-by":"crossref","first-page":"630","DOI":"10.1038\/gt.2017.71","article-title":"AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington\u2019s disease","volume":"24","author":"Miniarikova","year":"2017","journal-title":"Gene Ther."},{"key":"ref_178","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1016\/j.ymthe.2016.11.010","article-title":"CRISPR\/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo","volume":"25","author":"Monteys","year":"2017","journal-title":"Mol. Ther."},{"key":"ref_179","doi-asserted-by":"crossref","first-page":"2719","DOI":"10.1172\/JCI92087","article-title":"CRISPR\/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington\u2019s disease","volume":"127","author":"Yang","year":"2017","journal-title":"J. Clin. Investig."},{"key":"ref_180","doi-asserted-by":"crossref","first-page":"2163","DOI":"10.1016\/j.ymthe.2018.06.021","article-title":"AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington\u2019s Disease Minipig Model","volume":"26","author":"Evers","year":"2018","journal-title":"Mol. Ther."},{"key":"ref_181","doi-asserted-by":"crossref","first-page":"829","DOI":"10.1016\/j.omtn.2019.07.009","article-title":"CRISPR-Cas9-Mediated Genome Editing Increases Lifespan and Improves Motor Deficits in a Huntington\u2019s Disease Mouse Model","volume":"17","author":"Ekman","year":"2019","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_182","doi-asserted-by":"crossref","first-page":"334","DOI":"10.1016\/j.omtm.2019.03.002","article-title":"AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models","volume":"13","author":"Spronck","year":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"ref_183","first-page":"36","article-title":"Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease","volume":"48","author":"Caron","year":"2020","journal-title":"Nucleic Acids Res."},{"key":"ref_184","unstructured":"(2024, August 28). Voyager Therapeutics Announces Preclinical Data for Huntington\u2019s Disease and Amyotrophic Lateral Sclerosis Programs at the Congress of the European Society of Gene and Cell Therapy. Available online: https:\/\/ir.voyagertherapeutics.com\/news-releases\/news-release-details\/voyager-therapeutics-announces-preclinical-data-huntingtons\/."},{"key":"ref_185","unstructured":"(2024, September 02). Voyager Therapeutics Provides Regulatory Update on VY-HTT01 Program. Voyager Therapeutics Inc. Available online: https:\/\/ir.voyagertherapeutics.com\/news-releases\/news-release-details\/voyager-therapeutics-provides-regulatory-update-vy-htt01-program\/."},{"key":"ref_186","doi-asserted-by":"crossref","unstructured":"Spronck, E.A., Vall\u00e8s, A., Lampen, M.H., Montenegro-Miranda, P.S., Keskin, S., Heijink, L., Evers, M.M., Petry, H., Deventer, S.J.V., and Konstantinova, P. (2021). Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology. Brain Sci., 11.","DOI":"10.3390\/brainsci11020129"},{"key":"ref_187","doi-asserted-by":"crossref","first-page":"eabb8920","DOI":"10.1126\/scitranslmed.abb8920","article-title":"Widespread and sustained target engagement in Huntington\u2019s disease minipigs upon intrastriatal microRNA-based gene therapy","volume":"13","author":"Evers","year":"2021","journal-title":"Sci. Transl. Med."},{"key":"ref_188","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1089\/hum.2021.221","article-title":"Efficient and Precise Processing of the Optimized Primary Artificial MicroRNA in a Huntingtin-Lowering Adeno-Associated Viral Gene Therapy In Vitro and in Mice and Nonhuman Primates","volume":"33","author":"Wang","year":"2022","journal-title":"Hum. Gene Ther."},{"key":"ref_189","doi-asserted-by":"crossref","first-page":"2298","DOI":"10.1093\/brain\/awac458","article-title":"AAV5-miHTT-mediated huntingtin lowering improves brain health in a Huntington\u2019s disease mouse model","volume":"146","author":"Thomson","year":"2023","journal-title":"Brain"},{"key":"ref_190","doi-asserted-by":"crossref","first-page":"e202302018","DOI":"10.26508\/lsa.202302018","article-title":"Neuroprotective effects of hepatoma-derived growth factor in models of Huntington\u2019s disease","volume":"6","author":"Voelkl","year":"2023","journal-title":"Life Sci. Alliance"},{"key":"ref_191","doi-asserted-by":"crossref","first-page":"4043","DOI":"10.1093\/brain\/awae266","article-title":"Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington disease models","volume":"147","author":"Landles","year":"2024","journal-title":"Brain"},{"key":"ref_192","unstructured":"(2024, September 02). UCSF Huntington\u2019s Disease Trial: (POC) Study with AMT-130 in Adults with Early Manifest Huntington\u2019s Disease. Available online: https:\/\/clinicaltrials.ucsf.edu\/trial\/NCT04120493."},{"key":"ref_193","doi-asserted-by":"crossref","first-page":"351","DOI":"10.3233\/JHD-229006","article-title":"Huntington\u2019s Disease Clinical Trials Corner: November 2022","volume":"11","author":"Tabrizi","year":"2022","journal-title":"J. Huntington\u2019s Dis."},{"key":"ref_194","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1016\/S0014-4886(03)00044-X","article-title":"Structural and functional neuroprotection in a rat model of Huntington\u2019s disease by viral gene transfer of GDNF","volume":"181","author":"McBride","year":"2003","journal-title":"Exp. Neurol."},{"key":"ref_195","doi-asserted-by":"crossref","first-page":"682","DOI":"10.1016\/j.ymthe.2004.02.016","article-title":"AAV-mediated gene delivery of BDNF or GDNF is neuroprotective in a model of Huntington disease","volume":"9","author":"Kells","year":"2004","journal-title":"Mol. Ther."},{"key":"ref_196","doi-asserted-by":"crossref","first-page":"5868","DOI":"10.1073\/pnas.0801775105","article-title":"Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi","volume":"105","author":"McBride","year":"2008","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_197","doi-asserted-by":"crossref","first-page":"40","DOI":"10.1016\/j.nbd.2008.12.005","article-title":"Intrastriatal CERE-120 (AAV-Neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of Huntington\u2019s disease","volume":"34","author":"Ramaswamy","year":"2009","journal-title":"Neurobiol. Dis."},{"key":"ref_198","doi-asserted-by":"crossref","first-page":"13589","DOI":"10.1523\/JNEUROSCI.4286-09.2009","article-title":"Intrabody gene therapy ameliorates motor, cognitive, and neuropathological symptoms in multiple mouse models of Huntington\u2019s disease","volume":"29","author":"Southwell","year":"2009","journal-title":"J. Neurosci."},{"key":"ref_199","doi-asserted-by":"crossref","first-page":"428","DOI":"10.1016\/j.cmet.2011.02.013","article-title":"Mutant huntingtin causes metabolic imbalance by disruption of hypothalamic neurocircuits","volume":"13","author":"Hult","year":"2011","journal-title":"Cell Metab."},{"key":"ref_200","doi-asserted-by":"crossref","first-page":"2789","DOI":"10.1111\/j.1460-9568.2012.08193.x","article-title":"Characterization of a rat model of Huntington\u2019s disease based on targeted expression of mutant huntingtin in the forebrain using adeno-associated viral vectors","volume":"36","author":"Gabery","year":"2012","journal-title":"Eur. J. Neurosci."},{"key":"ref_201","doi-asserted-by":"crossref","first-page":"558","DOI":"10.1016\/j.bbrc.2012.03.033","article-title":"AAV-mediated delivery of the transcription factor XBP1s into the striatum reduces mutant Huntingtin aggregation in a mouse model of Huntington\u2019s disease","volume":"420","author":"Zuleta","year":"2012","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"ref_202","doi-asserted-by":"crossref","first-page":"787","DOI":"10.1016\/j.stem.2013.04.014","article-title":"Sustained mobilization of endogenous neural progenitors delays disease progression in a transgenic model of Huntington\u2019s disease","volume":"12","author":"Benraiss","year":"2013","journal-title":"Cell Stem Cell"},{"key":"ref_203","doi-asserted-by":"crossref","first-page":"e234","DOI":"10.1038\/mtna.2015.7","article-title":"Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo","volume":"4","author":"Monteys","year":"2015","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"ref_204","doi-asserted-by":"crossref","first-page":"726","DOI":"10.1038\/mt.2015.231","article-title":"Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector","volume":"24","author":"Choudhury","year":"2016","journal-title":"Mol. Ther."},{"key":"ref_205","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1038\/gt.2015.113","article-title":"AAV1\/2-mediated BDNF gene therapy in a transgenic rat model of Huntington\u2019s disease","volume":"23","author":"Connor","year":"2016","journal-title":"Gene Ther."},{"key":"ref_206","doi-asserted-by":"crossref","first-page":"239","DOI":"10.3233\/JHD-160215","article-title":"Cellular Analysis of Silencing the Huntington\u2019s Disease Gene Using AAV9 Mediated Delivery of Artificial Micro RNA into the Striatum of Q140\/Q140 Mice","volume":"5","author":"Keeler","year":"2016","journal-title":"J. Huntington\u2019s Dis."},{"key":"ref_207","doi-asserted-by":"crossref","first-page":"663","DOI":"10.1089\/hum.2017.199","article-title":"Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington\u2019s Disease","volume":"29","author":"Pfister","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"ref_208","doi-asserted-by":"crossref","first-page":"20265","DOI":"10.1073\/pnas.2002283117","article-title":"Loss of Hap1 selectively promotes striatal degeneration in Huntington disease mice","volume":"117","author":"Liu","year":"2020","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_209","doi-asserted-by":"crossref","first-page":"104951","DOI":"10.1016\/j.nbd.2020.104951","article-title":"Use of adeno-associated virus-mediated delivery of mutant huntingtin to study the spreading capacity of the protein in mice and non-human primates","volume":"141","author":"Maxan","year":"2020","journal-title":"Neurobiol. Dis."},{"key":"ref_210","doi-asserted-by":"crossref","first-page":"3175","DOI":"10.1093\/brain\/awab186","article-title":"SREBP2 gene therapy targeting striatal astrocytes ameliorates Huntington\u2019s disease phenotypes","volume":"144","author":"Birolini","year":"2021","journal-title":"Brain"},{"key":"ref_211","doi-asserted-by":"crossref","first-page":"102110","DOI":"10.1016\/j.pneurobio.2021.102110","article-title":"Dysfunction of X-linked inhibitor of apoptosis protein (XIAP) triggers neuropathological processes via altered p53 activity in Huntington\u2019s disease","volume":"204","author":"Hyeon","year":"2021","journal-title":"Prog. Neurobiol."},{"key":"ref_212","doi-asserted-by":"crossref","first-page":"185","DOI":"10.3233\/JHD-200002","article-title":"Huntington\u2019s Disease Clinical Trials Corner: April 2020","volume":"9","author":"Rodrigues","year":"2020","journal-title":"J. Huntington\u2019s Dis."},{"key":"ref_213","unstructured":"(2024, September 02). uniQure Announces Completion of Enrollment in First Cohort of Phase I\/II Clinical Trial of AMT-130 for the Treatment of Huntington\u2019s Disease. Available online: https:\/\/www.uniqure.com\/programs-pipeline\/phase-1-2-clinical-trial-of-amt-130."},{"key":"ref_214","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1007\/s40259-022-00519-9","article-title":"Antisense Oligonucleotide Therapy: From Design to the Huntington Disease Clinic","volume":"36","author":"Rook","year":"2022","journal-title":"BioDrugs"},{"key":"ref_215","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1016\/j.nbd.2015.11.019","article-title":"Recombinant Adeno Associated Viral (AAV) vector type 9 delivery of Ex1-Q138-mutant huntingtin in the rat striatum as a short-time model for in vivo studies in drug discovery","volume":"86","author":"Ceccarelli","year":"2016","journal-title":"Neurobiol. Dis."},{"key":"ref_216","doi-asserted-by":"crossref","first-page":"157","DOI":"10.3389\/fncel.2018.00157","article-title":"Adeno-Associated Viral Vector Serotype DJ-Mediated Overexpression of N171-82Q-Mutant Huntingtin in the Striatum of Juvenile Mice Is a New Model for Huntington\u2019s Disease","volume":"12","author":"Jang","year":"2018","journal-title":"Front. Cell Neurosci."},{"key":"ref_217","doi-asserted-by":"crossref","first-page":"579","DOI":"10.3340\/jkns.2019.0182","article-title":"An Optimization of AAV-82Q-Delivered Rat Model of Huntington\u2019s Disease","volume":"63","author":"So","year":"2020","journal-title":"J. Korean Neurosurg. Soc."},{"key":"ref_218","doi-asserted-by":"crossref","first-page":"1316","DOI":"10.1080\/15548627.2020.1760014","article-title":"Impact of differential and time-dependent autophagy activation on therapeutic efficacy in a model of Huntington disease","volume":"17","author":"Hersbach","year":"2021","journal-title":"Autophagy"},{"key":"ref_219","doi-asserted-by":"crossref","first-page":"811","DOI":"10.1016\/0092-8674(95)90542-1","article-title":"Targeted disruption of the Huntington\u2019s disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes","volume":"81","author":"Nasir","year":"1995","journal-title":"Cell"},{"key":"ref_220","doi-asserted-by":"crossref","first-page":"155","DOI":"10.1038\/ng1095-155","article-title":"Increased apoptosis and early embryonic lethality in mice nullizygous for the Huntington\u2019s disease gene homologue","volume":"11","author":"Zeitlin","year":"1995","journal-title":"Nat. Genet."},{"key":"ref_221","doi-asserted-by":"crossref","first-page":"300","DOI":"10.1038\/81593","article-title":"Inactivation of Hdh in the brain and testis results in progressive neurodegeneration and sterility in mice","volume":"26","author":"Dragatsis","year":"2000","journal-title":"Nat. Genet."},{"key":"ref_222","doi-asserted-by":"crossref","first-page":"764","DOI":"10.1001\/jamaneurol.2020.0299","article-title":"Huntingtin-Lowering Therapies for Huntington Disease: A Review of the Evidence of Potential Benefits and Risks","volume":"77","author":"Leavitt","year":"2020","journal-title":"JAMA Neurol."}],"container-title":["Cells"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/2073-4409\/14\/19\/1514\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,9,29]],"date-time":"2025-09-29T11:10:11Z","timestamp":1759144211000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/2073-4409\/14\/19\/1514"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,9,28]]},"references-count":222,"journal-issue":{"issue":"19","published-online":{"date-parts":[[2025,10]]}},"alternative-id":["cells14191514"],"URL":"https:\/\/doi.org\/10.3390\/cells14191514","relation":{},"ISSN":["2073-4409"],"issn-type":[{"value":"2073-4409","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,9,28]]}}}