{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T10:14:39Z","timestamp":1792059279013,"version":"4.4.0"},"reference-count":168,"publisher":"Elsevier BV","issue":"16","license":[{"start":{"date-parts":[[2025,8,1]],"date-time":"2025-08-01T00:00:00Z","timestamp":1754006400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,8,1]],"date-time":"2025-08-01T00:00:00Z","timestamp":1754006400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,7,2]],"date-time":"2025-07-02T00:00:00Z","timestamp":1751414400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100008178","name":"Davis Family Foundation","doi-asserted-by":"publisher","award":["RM1HG009490"],"award-info":[{"award-number":["RM1HG009490"]}],"id":[{"id":"10.13039\/100008178","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008178","name":"Davis Family Foundation","doi-asserted-by":"publisher","award":["R35GM118062"],"award-info":[{"award-number":["R35GM118062"]}],"id":[{"id":"10.13039\/100008178","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000011","name":"Howard Hughes Medical Institute","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000011","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["NS058949"],"award-info":[{"award-number":["NS058949"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["2022-316747"],"award-info":[{"award-number":["2022-316747"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["2024-347822"],"award-info":[{"award-number":["2024-347822"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000001","name":"National Science Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000001","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100014989","name":"The Chan Zuckerberg Initiative","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014989","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Cell"],"published-print":{"date-parts":[[2025,8]]},"DOI":"10.1016\/j.cell.2025.06.038","type":"journal-article","created":{"date-parts":[[2025,7,21]],"date-time":"2025-07-21T14:37:46Z","timestamp":1753108666000},"page":"4275-4294.e23","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":53,"title":["In vivo prime editing rescues alternating hemiplegia of childhood in mice"],"prefix":"10.1016","volume":"188","author":[{"given":"Alexander A.","family":"Sousa","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Markus","family":"Terrey","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Holt A.","family":"Sakai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine Q.","family":"Simmons","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena","family":"Arystarkhova","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalia S.","family":"Morsci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura C.","family":"Anderson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jun","family":"Xie","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fabian","family":"Suri-Payer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linda C.","family":"Laux","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emmanuel","family":"Roze","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvie","family":"Forlani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guangping","family":"Gao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simon","family":"Frost","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nina","family":"Frost","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen J.","family":"Sweadner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"suffix":"Jr.","given":"Alfred L.","family":"George","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cathleen M.","family":"Lutz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9943-7557","authenticated-orcid":false,"given":"David R.","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.cell.2025.06.038_bib1","doi-asserted-by":"crossref","DOI":"10.1007\/s11940-017-0444-7","article-title":"Diagnosis and Treatment of Alternating Hemiplegia of Childhood","volume":"19","author":"Masoud","year":"2017","journal-title":"Curr. Treat. Options Neurol."},{"key":"10.1016\/j.cell.2025.06.038_bib2","doi-asserted-by":"crossref","first-page":"777","DOI":"10.1111\/j.1469-8749.2007.00777.x","article-title":"The treatment and management of alternating hemiplegia of childhood","volume":"49","author":"Neville","year":"2007","journal-title":"Dev. Med. Child Neurol."},{"key":"10.1016\/j.cell.2025.06.038_bib3","doi-asserted-by":"crossref","first-page":"1030","DOI":"10.1038\/ng.2358","article-title":"De novo mutations in ATP1A3 cause alternating hemiplegia of childhood","volume":"44","author":"Heinzen","year":"2012","journal-title":"Nat. Genet."},{"key":"10.1016\/j.cell.2025.06.038_bib4","doi-asserted-by":"crossref","DOI":"10.1186\/s13023-015-0335-5","article-title":"Clinical profile of patients with ATP1A3 mutations in Alternating Hemiplegia of Childhood\u2014a study of 155 patients","volume":"10","author":"Panagiotakaki","year":"2015","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.cell.2025.06.038_bib5","doi-asserted-by":"crossref","first-page":"764","DOI":"10.1016\/S1474-4422(12)70182-5","article-title":"Heterozygous de-novo mutations in ATP1A3 in patients with alternating hemiplegia of childhood: a whole-exome sequencing gene-identification study","volume":"11","author":"Rosewich","year":"2012","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.cell.2025.06.038_bib6","doi-asserted-by":"crossref","first-page":"e1511","DOI":"10.1212\/WNL.0000000000200927","article-title":"The Phenotypic Continuum of ATP1A3-Related Disorders","volume":"99","author":"Vezyroglou","year":"2022","journal-title":"Neurology"},{"key":"10.1016\/j.cell.2025.06.038_bib7","doi-asserted-by":"crossref","first-page":"1807","DOI":"10.1016\/j.bbabio.2016.08.009","article-title":"Neurological disease mutations of \u03b13 Na+,K+-ATPase: Structural and functional perspectives and rescue of compromised function","volume":"1857","author":"Holm","year":"2016","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.cell.2025.06.038_bib8","doi-asserted-by":"crossref","first-page":"503","DOI":"10.1016\/S1474-4422(14)70011-0","article-title":"Distinct neurological disorders with ATP1A3 mutations","volume":"13","author":"Heinzen","year":"2014","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.cell.2025.06.038_bib9","doi-asserted-by":"crossref","first-page":"482","DOI":"10.1212\/WNL.0000000000000102","article-title":"Genotype\u2013phenotype correlations in alternating hemiplegia of childhood","volume":"82","author":"Sasaki","year":"2014","journal-title":"Neurology"},{"key":"10.1016\/j.cell.2025.06.038_bib10","article-title":"ATP1A3 Mutations and Genotype-Phenotype Correlation of Alternating Hemiplegia of Childhood in Chinese Patients","volume":"9","author":"Yang","year":"2014","journal-title":"PLOS One"},{"key":"10.1016\/j.cell.2025.06.038_bib11","article-title":"Alternating Hemiplegia of Childhood: Retrospective Genetic Study and Genotype-Phenotype Correlations in 187 Subjects from the US AHCF Registry","volume":"10","author":"Viollet","year":"2015","journal-title":"PLOS One"},{"key":"10.1016\/j.cell.2025.06.038_bib12","doi-asserted-by":"crossref","first-page":"945","DOI":"10.1212\/WNL.0000000000000212","article-title":"The expanding clinical and genetic spectrum of ATP1A3-related disorders","volume":"82","author":"Rosewich","year":"2014","journal-title":"Neurology"},{"key":"10.1016\/j.cell.2025.06.038_bib13","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1016\/j.nbd.2015.02.002","article-title":"A functional correlate of severity in alternating hemiplegia of childhood","volume":"77","author":"Li","year":"2015","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.cell.2025.06.038_bib14","doi-asserted-by":"crossref","DOI":"10.3389\/fphys.2016.00209","article-title":"Insights into the Pathology of the \u03b13 Na(+)\/K(+)-ATPase Ion Pump in Neurological Disorders; Lessons from Animal Models","volume":"7","author":"Holm","year":"2016","journal-title":"Front. Physiol."},{"key":"10.1016\/j.cell.2025.06.038_bib15","doi-asserted-by":"crossref","first-page":"14085","DOI":"10.1073\/pnas.0904817106","article-title":"Mutation I810N in the \u03b13 isoform of Na+,K+-ATPase causes impairments in the sodium pump and hyperexcitability in the CNS","volume":"106","author":"Clapcote","year":"2009","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.cell.2025.06.038_bib16","doi-asserted-by":"crossref","first-page":"3433","DOI":"10.1113\/jphysiol.2012.247817","article-title":"Enhanced inhibitory neurotransmission in the cerebellar cortex of Atp1a3-deficient heterozygous mice","volume":"591","author":"Ikeda","year":"2013","journal-title":"J. Physiol."},{"key":"10.1016\/j.cell.2025.06.038_bib17","doi-asserted-by":"crossref","first-page":"100","DOI":"10.1016\/j.bbr.2014.06.048","article-title":"Heterozygous mice deficient in Atp1a3 exhibit motor deficits by chronic restraint stress","volume":"272","author":"Sugimoto","year":"2014","journal-title":"Behav. Brain Res."},{"key":"10.1016\/j.cell.2025.06.038_bib18","doi-asserted-by":"crossref","first-page":"82","DOI":"10.1111\/epi.12878","article-title":"Knock-in mouse model of alternating hemiplegia of childhood: Behavioral and electrophysiologic characterization","volume":"56","author":"Hunanyan","year":"2015","journal-title":"Epilepsia"},{"key":"10.1016\/j.cell.2025.06.038_bib19","doi-asserted-by":"crossref","first-page":"100","DOI":"10.1016\/j.nbd.2018.07.028","article-title":"Novel E815K knock-in mouse model of alternating hemiplegia of childhood","volume":"119","author":"Helseth","year":"2018","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.cell.2025.06.038_bib20","doi-asserted-by":"crossref","first-page":"616","DOI":"10.1523\/JNEUROSCI.4464-06.2007","article-title":"Deficiency in Na,K-ATPase \u03b1 Isoform Genes Alters Spatial Learning, Motor Activity, and Anxiety in Mice","volume":"27","author":"Moseley","year":"2007","journal-title":"J. Neurosci."},{"key":"10.1016\/j.cell.2025.06.038_bib21","doi-asserted-by":"crossref","first-page":"659","DOI":"10.1016\/j.bbr.2010.09.009","article-title":"Characterization of Atp1a3 mutant mice as a model of rapid-onset dystonia with parkinsonism","volume":"216","author":"DeAndrade","year":"2011","journal-title":"Behav. Brain Res."},{"key":"10.1016\/j.cell.2025.06.038_bib22","doi-asserted-by":"crossref","DOI":"10.1523\/ENEURO.0101-24.2024","article-title":"Phenotype Distinctions in Mice Deficient in the Neuron-Specific \u03b13 Subunit of Na,K-ATPase: Atp1a3tm1Ling\/+ and Atp1a3+\/D801Y","volume":"11","author":"Liu","year":"2024","journal-title":"eNeuro"},{"key":"10.1016\/j.cell.2025.06.038_bib23","doi-asserted-by":"crossref","DOI":"10.1016\/j.nbd.2024.106751","article-title":"Novel mouse model of alternating hemiplegia of childhood exhibits prominent motor and seizure phenotypes","volume":"203","author":"Hawkins","year":"2024","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.cell.2025.06.038_bib24","doi-asserted-by":"crossref","first-page":"405","DOI":"10.1089\/hum.2020.191","article-title":"Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3Mashl\/+ , Mouse Model of Alternating Hemiplegia of Childhood","volume":"32","author":"Hunanyan","year":"2021","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.cell.2025.06.038_bib25","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1016\/j.nbd.2018.03.009","article-title":"Direct evidence of impaired neuronal Na\/K-ATPase pump function in alternating hemiplegia of childhood","volume":"115","author":"Simmons","year":"2018","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.cell.2025.06.038_bib26","doi-asserted-by":"crossref","first-page":"824","DOI":"10.1038\/s41587-020-0561-9","article-title":"Genome editing with CRISPR\u2013Cas nucleases, base editors, transposases and prime editors","volume":"38","author":"Anzalone","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib27","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1038\/s41576-022-00541-1","article-title":"Prime editing for precise and highly versatile genome manipulation","volume":"24","author":"Chen","year":"2023","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.cell.2025.06.038_bib28","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1038\/s41580-023-00697-6","article-title":"CRISPR technologies for genome, epigenome and transcriptome editing","volume":"25","author":"Villiger","year":"2024","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.cell.2025.06.038_bib29","doi-asserted-by":"crossref","first-page":"3107","DOI":"10.1016\/j.ymthe.2021.09.002","article-title":"In vivo somatic cell base editing and prime editing","volume":"29","author":"Newby","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.cell.2025.06.038_bib30","doi-asserted-by":"crossref","first-page":"770","DOI":"10.1038\/s41576-018-0059-1","article-title":"Base editing: precision chemistry on the genome and transcriptome of living cells","volume":"19","author":"Rees","year":"2018","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.cell.2025.06.038_bib31","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1038\/nbt.4192","article-title":"Repair of double-strand breaks induced by CRISPR\u2013Cas9 leads to large deletions and complex rearrangements","volume":"36","author":"Kosicki","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib32","doi-asserted-by":"crossref","first-page":"523","DOI":"10.1016\/j.omtn.2020.06.019","article-title":"Large-Fragment Deletions Induced by Cas9 Cleavage while Not in the BEs System","volume":"21","author":"Song","year":"2020","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.cell.2025.06.038_bib33","doi-asserted-by":"crossref","DOI":"10.1073\/pnas.2004832117","article-title":"Frequent loss of heterozygosity in CRISPR-Cas9\u2013edited early human embryos","volume":"118","author":"Alanis-Lobato","year":"2021","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.cell.2025.06.038_bib34","doi-asserted-by":"crossref","first-page":"1650","DOI":"10.1016\/j.cell.2020.10.025","article-title":"Allele-Specific Chromosome Removal after Cas9 Cleavage in Human Embryos","volume":"183","author":"Zuccaro","year":"2020","journal-title":"Cell"},{"key":"10.1016\/j.cell.2025.06.038_bib35","doi-asserted-by":"crossref","first-page":"895","DOI":"10.1038\/s41588-021-00838-7","article-title":"Chromothripsis as an on-target consequence of CRISPR\u2013Cas9 genome editing","volume":"53","author":"Leibowitz","year":"2021","journal-title":"Nat. Genet."},{"key":"10.1016\/j.cell.2025.06.038_bib36","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-019-09006-2","article-title":"CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations","volume":"10","author":"Cullot","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.cell.2025.06.038_bib37","doi-asserted-by":"crossref","first-page":"3685","DOI":"10.1038\/s41467-022-31322-3","article-title":"Frequency and mechanisms of LINE-1 retrotransposon insertions at CRISPR\/Cas9 sites","volume":"13","author":"Tao","year":"2022","journal-title":"Nat. Commun."},{"key":"10.1016\/j.cell.2025.06.038_bib38","doi-asserted-by":"crossref","first-page":"927","DOI":"10.1038\/s41591-018-0049-z","article-title":"CRISPR\u2013Cas9 genome editing induces a p53-mediated DNA damage response","volume":"24","author":"Haapaniemi","year":"2018","journal-title":"Nat. Med."},{"key":"10.1016\/j.cell.2025.06.038_bib39","doi-asserted-by":"crossref","first-page":"662","DOI":"10.1038\/s41588-020-0623-4","article-title":"Cas9 activates the p53 pathway and selects for p53-inactivating mutations","volume":"52","author":"Enache","year":"2020","journal-title":"Nat. Genet."},{"key":"10.1016\/j.cell.2025.06.038_bib40","doi-asserted-by":"crossref","first-page":"939","DOI":"10.1038\/s41591-018-0050-6","article-title":"p53 inhibits CRISPR\u2013Cas9 engineering in human pluripotent stem cells","volume":"24","author":"Ihry","year":"2018","journal-title":"Nat. Med."},{"key":"10.1016\/j.cell.2025.06.038_bib41","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1038\/nature17946","article-title":"Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage","volume":"533","author":"Komor","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.cell.2025.06.038_bib42","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1038\/nature24644","article-title":"Programmable base editing of A\u00b7T to G\u00b7C in genomic DNA without DNA cleavage","volume":"551","author":"Gaudelli","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.cell.2025.06.038_bib43","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"key":"10.1016\/j.cell.2025.06.038_bib44","doi-asserted-by":"crossref","first-page":"2431","DOI":"10.1038\/s41596-022-00724-4","article-title":"Designing and executing prime editing experiments in mammalian cells","volume":"17","author":"Doman","year":"2022","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.cell.2025.06.038_bib45","doi-asserted-by":"crossref","first-page":"1089","DOI":"10.1038\/s41596-020-00450-9","article-title":"Precision genome editing using cytosine and adenine base editors in mammalian cells","volume":"16","author":"Huang","year":"2021","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.cell.2025.06.038_bib46","doi-asserted-by":"crossref","first-page":"1343","DOI":"10.1016\/j.ymthe.2022.01.005","article-title":"A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver","volume":"30","author":"Zheng","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.cell.2025.06.038_bib47","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1016\/j.ymthe.2021.07.011","article-title":"Dual-AAV delivering split prime editor system for in vivo genome editing","volume":"30","author":"Zhi","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.cell.2025.06.038_bib48","article-title":"Dual-AAV split prime editor corrects the mutation and phenotype in mice with inherited retinal degeneration","volume":"8","author":"She","year":"2023","journal-title":"Signal Transduct. Target. Ther."},{"key":"10.1016\/j.cell.2025.06.038_bib49","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.abl9238","article-title":"In vivo prime editing of a metabolic liver disease in mice","volume":"14","author":"B\u00f6ck","year":"2022","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.cell.2025.06.038_bib50","doi-asserted-by":"crossref","first-page":"2942","DOI":"10.1016\/j.ymthe.2022.07.001","article-title":"A truncated reverse transcriptase enhances prime editing by split AAV vectors","volume":"30","author":"Gao","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.cell.2025.06.038_bib51","article-title":"Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice","volume":"12","author":"Liu","year":"2021","journal-title":"Nat. Commun."},{"key":"10.1016\/j.cell.2025.06.038_bib52","doi-asserted-by":"crossref","first-page":"3983","DOI":"10.1016\/j.cell.2023.07.039","article-title":"Phage-assisted evolution and protein engineering yield compact, efficient prime editors","volume":"186","author":"Doman","year":"2023","journal-title":"Cell"},{"key":"10.1016\/j.cell.2025.06.038_bib53","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1038\/s41587-023-01758-z","article-title":"Efficient prime editing in mouse brain, liver and heart with dual AAVs","volume":"42","author":"Davis","year":"2024","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib54","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1038\/s41551-021-00788-9","article-title":"Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases","volume":"6","author":"Jang","year":"2022","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.cell.2025.06.038_bib55","doi-asserted-by":"crossref","first-page":"1526","DOI":"10.1038\/s41587-023-02078-y","article-title":"Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo","volume":"42","author":"An","year":"2024","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib56","doi-asserted-by":"crossref","first-page":"1319","DOI":"10.1038\/s41591-024-03466-w","article-title":"In vivo base editing extends lifespan of a humanized mouse model of prion disease","volume":"31","author":"An","year":"2025","journal-title":"Nat. Med."},{"key":"10.1016\/j.cell.2025.06.038_bib57","doi-asserted-by":"crossref","DOI":"10.1126\/science.adg6518","article-title":"Base editing rescue of spinal muscular atrophy in cells and in mice","volume":"380","author":"Arbab","year":"2023","journal-title":"Science"},{"key":"10.1016\/j.cell.2025.06.038_bib58","year":"2023"},{"key":"10.1016\/j.cell.2025.06.038_bib59","doi-asserted-by":"crossref","first-page":"250","DOI":"10.1016\/j.cell.2021.12.021","article-title":"Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins","volume":"185","author":"Banskota","year":"2022","journal-title":"Cell"},{"key":"10.1016\/j.cell.2025.06.038_bib60","doi-asserted-by":"crossref","first-page":"429","DOI":"10.1038\/s41586-021-03534-y","article-title":"In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates","volume":"593","author":"Musunuru","year":"2021","journal-title":"Nature"},{"key":"10.1016\/j.cell.2025.06.038_bib61","doi-asserted-by":"crossref","first-page":"242","DOI":"10.1161\/CIRCULATIONAHA.122.062132","article-title":"Efficacy and Safety of an Investigational Single-Course CRISPR Base-Editing Therapy Targeting PCSK9 in Nonhuman Primate and Mouse Models","volume":"147","author":"Lee","year":"2023","journal-title":"Circulation"},{"key":"10.1016\/j.cell.2025.06.038_bib62","doi-asserted-by":"crossref","first-page":"295","DOI":"10.1038\/s41586-021-03609-w","article-title":"Base editing of haematopoietic stem cells rescues sickle cell disease in mice","volume":"595","author":"Newby","year":"2021","journal-title":"Nature"},{"key":"10.1016\/j.cell.2025.06.038_bib63","doi-asserted-by":"crossref","first-page":"608","DOI":"10.1038\/s41586-020-03086-7","article-title":"In vivo base editing rescues Hutchinson\u2013Gilford progeria syndrome in mice","volume":"589","author":"Koblan","year":"2021","journal-title":"Nature"},{"key":"10.1016\/j.cell.2025.06.038_bib64","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1038\/s41551-024-01296-2","article-title":"Safer and efficient base editing and prime editing via ribonucleoproteins delivered through optimized lipid-nanoparticle formulations","volume":"9","author":"Ho\u0142ubowicz","year":"2025","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.cell.2025.06.038_bib65","doi-asserted-by":"crossref","DOI":"10.1016\/j.celrep.2024.115144","article-title":"Improved split prime editors enable efficient in vivo genome editing","volume":"44","author":"Wei","year":"2025","journal-title":"Cell Rep."},{"key":"10.1016\/j.cell.2025.06.038_bib66","doi-asserted-by":"crossref","first-page":"1822","DOI":"10.1038\/s41587-023-02106-x","article-title":"Efficient prime editing in two-cell mouse embryos using PEmbryo","volume":"42","author":"Kim-Yip","year":"2024","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib67","first-page":"1","article-title":"Increasing intracellular dNTP levels improves prime editing efficiency","author":"Liu","year":"2024","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib68","doi-asserted-by":"crossref","first-page":"2003","DOI":"10.1016\/j.ajhg.2023.10.005","article-title":"Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need","volume":"110","author":"Brooks","year":"2023","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.cell.2025.06.038_bib69","article-title":"Template-jumping prime editing enables large insertion and exon rewriting in vivo","volume":"14","author":"Zheng","year":"2023","journal-title":"Nat. Commun."},{"key":"10.1016\/j.cell.2025.06.038_bib70","first-page":"2085","article-title":"In vivo HSC prime editing rescues sickle cell disease in a mouse model","volume":"141","author":"Li","year":"2023","journal-title":"Blood"},{"key":"10.1016\/j.cell.2025.06.038_bib71","doi-asserted-by":"crossref","DOI":"10.1084\/jem.20220776","article-title":"Vision rescue via unconstrained in vivo prime editing in degenerating neural retinas","volume":"220","author":"Qin","year":"2023","journal-title":"J. Exp. Med."},{"key":"10.1016\/j.cell.2025.06.038_bib72","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1038\/s41587-021-01026-y","article-title":"Deletion and replacement of long genomic sequences using prime editing","volume":"40","author":"Jiang","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib73","doi-asserted-by":"crossref","first-page":"1437","DOI":"10.1038\/s41588-025-02172-8","article-title":"Base editing of trinucleotide repeats that cause Huntington\u2019s disease and Friedreich\u2019s ataxia reduces somatic repeat expansions in patient cells and in mice","volume":"57","author":"Matuszek","year":"2025","journal-title":"Nat. Genet."},{"key":"10.1016\/j.cell.2025.06.038_bib74","unstructured":"Vafai, S.B., Gladding, P.A., Scott, R., Kerr, J., Taubel, J., Cegla, J., Barbir, M., Humphries, S.E., Karsten, V., Jensen, C.L., et al. (2023). Safety and Pharmacodynamic Effects of VERVE-101. Data presented at the American Heart Association Scientific Sessions 2023 (Philadelphia, PA). https:\/\/live-verve-therapeutics.pantheonsite.io\/sites\/default\/files\/2023-11\/Verve_AHA_2023_LBS_for%20website.pdf."},{"key":"10.1016\/j.cell.2025.06.038_bib75","unstructured":"Khera, A.V. (2024). Developing single-course gene editing medicines to treat cardiovascular disease. Data presented at the European Society of Cell and Gene Therapy 31st Annual Congress, Rome, Italy, 22 October, 2024. https:\/\/www.vervetx.com\/sites\/default\/files\/2024-10\/Khera_ESGCT%202024%20FINAL_0.pdf."},{"key":"10.1016\/j.cell.2025.06.038_bib76","doi-asserted-by":"crossref","unstructured":"Gupta, A.O., Sharma, A., Frangoul, H., Dalal, J., Kanter, J., Alavi, A., DiPersio, J., Eapen, M., Jaroscak, J.J., Ayala, E., et al. (2024). Initial Results from the BEACON Clinical Study: A Phase 1\/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease with Severe Vaso-Occlusive Crises. Data presented at the 66th American Society of Hematology Annual Meeting and Exposition (San Diego, California). https:\/\/beamtx.com\/media\/3uengrm0\/beam-101-ash_presentation_final-for-website.pdf.","DOI":"10.1182\/blood-2024-204888"},{"key":"10.1016\/j.cell.2025.06.038_bib77","unstructured":"Verve Therapeutics, Inc. (2024). Open-label, Phase 1b, Single-ascending Dose and Optional Re Dosing Study to Evaluate the Safety of VERVE-101 Administered to Patients with Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, and Uncontrolled Hypercholesterolemia. NCT05398029. https:\/\/clinicaltrials.gov\/study\/NCT05398029."},{"key":"10.1016\/j.cell.2025.06.038_bib78","unstructured":"Beam Therapeutics Inc. (2025). A Phase 1\/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients With Sickle Cell Disease and Severe Vaso-Occlusive Crises (Beacon Trial). NCT05456880. https:\/\/clinicaltrials.gov\/study\/NCT05456880."},{"key":"10.1016\/j.cell.2025.06.038_bib79","doi-asserted-by":"crossref","first-page":"2235","DOI":"10.1056\/NEJMoa2504747","article-title":"Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease","volume":"392","author":"Musunuru","year":"2025","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.cell.2025.06.038_bib80","series-title":"Participants with Autosomal Recessive Chronic Granulomatous Disease Due to Mutations in the NCF1 Gene. NCT06559176","article-title":"A Phase 1&2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Modified Ex Vivo Using Prime Editing","volume":"PM359","year":"2024"},{"key":"10.1016\/j.cell.2025.06.038_bib81","doi-asserted-by":"crossref","first-page":"eabd2368","DOI":"10.1126\/sciadv.abd2368","article-title":"De novo ATP1A3 variants cause polymicrogyria","volume":"7","author":"Miyatake","year":"2021","journal-title":"Sci. Adv."},{"key":"10.1016\/j.cell.2025.06.038_bib82","doi-asserted-by":"crossref","DOI":"10.1073\/pnas.2023333118","article-title":"Early role for a Na+,K+-ATPase (ATP1A3) in brain development","volume":"118","author":"Smith","year":"2021","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.cell.2025.06.038_bib83","series-title":"ZOLGENSMA\u00ae (Onasemnogene abeparvovec-xioi)","year":"2019"},{"key":"10.1016\/j.cell.2025.06.038_bib84","doi-asserted-by":"crossref","first-page":"463","DOI":"10.1016\/j.cell.2020.05.037","article-title":"Determinants of Base Editing Outcomes from Target Library Analysis and Machine Learning","volume":"182","author":"Arbab","year":"2020","journal-title":"Cell"},{"key":"10.1016\/j.cell.2025.06.038_bib85","doi-asserted-by":"crossref","first-page":"883","DOI":"10.1038\/s41587-020-0453-z","article-title":"Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity","volume":"38","author":"Richter","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib86","doi-asserted-by":"crossref","first-page":"7","DOI":"10.1038\/s41551-024-01233-3","article-title":"Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells","volume":"9","author":"Sousa","year":"2025","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.cell.2025.06.038_bib87","doi-asserted-by":"crossref","first-page":"402","DOI":"10.1038\/s41587-021-01039-7","article-title":"Engineered pegRNAs improve prime editing efficiency","volume":"40","author":"Nelson","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib88","doi-asserted-by":"crossref","first-page":"5635","DOI":"10.1016\/j.cell.2021.09.018","article-title":"Enhanced prime editing systems by manipulating cellular determinants of editing outcomes","volume":"184","author":"Chen","year":"2021","journal-title":"Cell"},{"key":"10.1016\/j.cell.2025.06.038_bib89","doi-asserted-by":"crossref","first-page":"e161","DOI":"10.1038\/mtna.2014.12","article-title":"Pol III Promoters to Express Small RNAs: Delineation of Transcription Initiation","volume":"3","author":"Ma","year":"2014","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.cell.2025.06.038_bib90","doi-asserted-by":"crossref","first-page":"6811","DOI":"10.1093\/nar\/gkaa477","article-title":"5\u2032 modifications to CRISPR-Cas9 gRNA can change the dynamics and size of R-loops and inhibit DNA cleavage","volume":"48","author":"Mullally","year":"2020","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.cell.2025.06.038_bib91","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-020-15021-5","article-title":"Blackjack mutations improve the on-target activities of increased fidelity variants of SpCas9 with 5\u2032G-extended sgRNAs","volume":"11","author":"Kulcs\u00e1r","year":"2020","journal-title":"Nat. Commun."},{"key":"10.1016\/j.cell.2025.06.038_bib92","doi-asserted-by":"crossref","first-page":"490","DOI":"10.1038\/nature16526","article-title":"High-fidelity CRISPR\u2013Cas9 nucleases with no detectable genome-wide off-target effects","volume":"529","author":"Kleinstiver","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.cell.2025.06.038_bib93","doi-asserted-by":"crossref","first-page":"639","DOI":"10.1038\/s41586-024-07259-6","article-title":"Improving prime editing with an endogenous small RNA-binding protein","volume":"628","author":"Yan","year":"2024","journal-title":"Nature"},{"key":"10.1016\/j.cell.2025.06.038_bib94","doi-asserted-by":"crossref","DOI":"10.1186\/s13059-021-02389-w","article-title":"Targeted mutagenesis in mouse cells and embryos using an enhanced prime editor","volume":"22","author":"Park","year":"2021","journal-title":"Genome Biol."},{"key":"10.1016\/j.cell.2025.06.038_bib95","doi-asserted-by":"crossref","first-page":"607","DOI":"10.1038\/nmeth.4278","article-title":"CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR\u2013Cas9 nuclease off-targets","volume":"14","author":"Tsai","year":"2017","journal-title":"Nat. Methods"},{"key":"10.1016\/j.cell.2025.06.038_bib96","doi-asserted-by":"crossref","DOI":"10.1186\/1472-6750-11-80","article-title":"RNase H-dependent PCR (rhPCR): improved specificity and single nucleotide polymorphism detection using blocked cleavable primers","volume":"11","author":"Dobosy","year":"2011","journal-title":"BMC Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib97","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1016\/j.omtm.2021.03.024","article-title":"CRISPAltRations: A validated cloud-based approach for interrogation of double-strand break repair mediated by CRISPR genome editing","volume":"21","author":"Kurgan","year":"2021","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.cell.2025.06.038_bib98","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1038\/s41421-019-0132-8","article-title":"Efficient generation of mouse models with the prime editing system","volume":"6","author":"Liu","year":"2020","journal-title":"Cell Discov."},{"key":"10.1016\/j.cell.2025.06.038_bib99","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-020-19136-7","article-title":"Prime editing for functional repair in patient-derived disease models","volume":"11","author":"Schene","year":"2020","journal-title":"Nat. Commun."},{"key":"10.1016\/j.cell.2025.06.038_bib100","doi-asserted-by":"crossref","DOI":"10.26508\/lsa.202000940","article-title":"Evaluating CRISPR-based prime editing for cancer modeling and CFTR repair in organoids","volume":"4","author":"Geurts","year":"2021","journal-title":"Life Sci. Alliance"},{"key":"10.1016\/j.cell.2025.06.038_bib101","doi-asserted-by":"crossref","DOI":"10.1186\/s13059-021-02304-3","article-title":"Prime editing in mice reveals the essentiality of a single base in driving tissue-specific gene expression","volume":"22","author":"Gao","year":"2021","journal-title":"Genome Biol."},{"key":"10.1016\/j.cell.2025.06.038_bib102","doi-asserted-by":"crossref","first-page":"494","DOI":"10.1016\/j.omtn.2021.06.020","article-title":"Modeling a cataract disorder in mice with prime editing","volume":"25","author":"Lin","year":"2021","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.cell.2025.06.038_bib103","doi-asserted-by":"crossref","first-page":"1187","DOI":"10.1093\/nar\/gkab1295","article-title":"Comprehensive analysis of prime editing outcomes in human embryonic stem cells","volume":"50","author":"Habib","year":"2022","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.cell.2025.06.038_bib104","doi-asserted-by":"crossref","first-page":"276","DOI":"10.1089\/crispr.2021.0080","article-title":"Genomic and Transcriptomic Analyses of Prime Editing Guide RNA\u2013Independent Off-Target Effects by Prime Editors","volume":"5","author":"Gao","year":"2022","journal-title":"CRISPR J."},{"key":"10.1016\/j.cell.2025.06.038_bib105","doi-asserted-by":"crossref","first-page":"898","DOI":"10.1038\/s41592-023-01859-2","article-title":"Genome-wide profiling of prime editor off-target sites in vitro and in vivo using PE-tag","volume":"20","author":"Liang","year":"2023","journal-title":"Nat. Methods"},{"key":"10.1016\/j.cell.2025.06.038_bib106","doi-asserted-by":"crossref","first-page":"877","DOI":"10.1038\/s41587-023-01915-4","article-title":"Genotoxic effects of base and prime editing in human hematopoietic stem cells","volume":"42","author":"Fiumara","year":"2024","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib107","doi-asserted-by":"crossref","first-page":"1292","DOI":"10.1038\/s41587-021-00891-x","article-title":"Genome-wide specificity of prime editors in plants","volume":"39","author":"Jin","year":"2021","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib108","doi-asserted-by":"crossref","first-page":"10576","DOI":"10.1093\/nar\/gkaa764","article-title":"Unbiased investigation of specificities of prime editing systems in human cells","volume":"48","author":"Kim","year":"2020","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.cell.2025.06.038_bib109","doi-asserted-by":"crossref","first-page":"616","DOI":"10.1038\/s41551-023-01026-0","article-title":"Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice","volume":"7","author":"Everette","year":"2023","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.cell.2025.06.038_bib110","doi-asserted-by":"crossref","DOI":"10.1016\/j.nbd.2025.106954","article-title":"Alternating hemiplegia of childhood associated mutations in Atp1a3 reveal diverse neurological alterations in mice","volume":"212","author":"Terrey","year":"2025","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.cell.2025.06.038_bib111","doi-asserted-by":"crossref","first-page":"290","DOI":"10.1126\/science.aba8853","article-title":"Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants","volume":"368","author":"Walton","year":"2020","journal-title":"Science"},{"key":"10.1016\/j.cell.2025.06.038_bib112","series-title":"LUXTURNA\u00ae (voretigene neparvovec-rzyl)","year":"2017"},{"key":"10.1016\/j.cell.2025.06.038_bib113","series-title":"ROCTAVIAN\u00ae (valoctocogene roxaparvovec-rvox)","year":"2023"},{"key":"10.1016\/j.cell.2025.06.038_bib114","series-title":"ELEVIDYS\u00ae (delandistrogene moxeparvovec-rokl)","year":"2024"},{"key":"10.1016\/j.cell.2025.06.038_bib115","series-title":"BEQVEZ\u00ae (Fidanacogene elaparvovec-dzkt)","year":"2024"},{"key":"10.1016\/j.cell.2025.06.038_bib116","series-title":"KEBILIDI\u00ae (eladocagene exuparvovec-tneq)","year":"2024"},{"key":"10.1016\/j.cell.2025.06.038_bib117","doi-asserted-by":"crossref","first-page":"317","DOI":"10.1007\/s40259-017-0234-5","article-title":"Adeno-Associated Virus (AAV) as a Vector for Gene Therapy","volume":"31","author":"Naso","year":"2017","journal-title":"BioDrugs"},{"key":"10.1016\/j.cell.2025.06.038_bib118","doi-asserted-by":"crossref","first-page":"80","DOI":"10.1038\/mt.2009.255","article-title":"Effect of Genome Size on AAV Vector Packaging","volume":"18","author":"Wu","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.cell.2025.06.038_bib119","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1016\/j.omtm.2020.10.011","article-title":"CNS Transduction Benefits of AAV-PHP.eB over AAV9 Are Dependent on Administration Route and Mouse Strain","volume":"19","author":"Mathiesen","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.cell.2025.06.038_bib120","series-title":"Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are \u2265 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory. NCT05089656","article-title":"A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101","year":"2025"},{"key":"10.1016\/j.cell.2025.06.038_bib121","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1038\/s41551-019-0501-5","article-title":"Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses","volume":"4","author":"Levy","year":"2020","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.cell.2025.06.038_bib122","doi-asserted-by":"crossref","DOI":"10.7554\/eLife.79135","article-title":"Functional abnormalities in the cerebellothalamic pathways in a mouse model of DYT25 dystonia","volume":"11","author":"A\u00efssa","year":"2022","journal-title":"eLife"},{"key":"10.1016\/j.cell.2025.06.038_bib123","doi-asserted-by":"crossref","first-page":"177","DOI":"10.1016\/j.nbd.2010.08.025","article-title":"Neurophysiology of dystonia: The role of inhibition","volume":"42","author":"Hallett","year":"2011","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.cell.2025.06.038_bib124","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1002\/mds.22196","article-title":"Dystonia associated with pontomesencephalic lesions","volume":"24","author":"Loher","year":"2009","journal-title":"Mov. Disord."},{"key":"10.1016\/j.cell.2025.06.038_bib125","doi-asserted-by":"crossref","first-page":"2499","DOI":"10.1093\/brain\/awn168","article-title":"The basal ganglia and cerebellum interact in the expression of dystonic movement","volume":"131","author":"Neychev","year":"2008","journal-title":"Brain"},{"key":"10.1016\/j.cell.2025.06.038_bib126","article-title":"The Anatomical Basis for Dystonia: The Motor Network Model","volume":"7","author":"Jinnah","year":"2017","journal-title":"Tremor Other Hyperkinet. Mov. (NY)"},{"key":"10.1016\/j.cell.2025.06.038_bib127","doi-asserted-by":"crossref","first-page":"1203","DOI":"10.1111\/ejn.12126","article-title":"Viral transduction of the neonatal brain delivers controllable genetic mosaicism for visualising and manipulating neuronal circuits in vivo","volume":"37","author":"Kim","year":"2013","journal-title":"Eur. J. Neurosci."},{"key":"10.1016\/j.cell.2025.06.038_bib128","article-title":"Intracerebroventricular viral injection of the neonatal mouse brain for persistent and widespread neuronal transduction","volume":"91","author":"Kim","year":"2014","journal-title":"J. Vis. Exp."},{"key":"10.1016\/j.cell.2025.06.038_bib129","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0067680","article-title":"Capsid serotype and timing of injection determines AAV transduction in the neonatal mice brain","volume":"8","author":"Chakrabarty","year":"2013","journal-title":"PLOS One"},{"key":"10.1016\/j.cell.2025.06.038_bib130","series-title":"P-Type ATPases: Methods and Protocols Methods in Molecular Biology","first-page":"89","article-title":"Colorimetric Assays of Na,K-ATPase","author":"Sweadner","year":"2016"},{"key":"10.1016\/j.cell.2025.06.038_bib131","doi-asserted-by":"crossref","DOI":"10.3389\/fnins.2021.689938","article-title":"Neurofilament Proteins as Biomarkers to Monitor Neurological Diseases and the Efficacy of Therapies","volume":"15","author":"Yuan","year":"2021","journal-title":"Front. Neurosci."},{"key":"10.1016\/j.cell.2025.06.038_bib132","doi-asserted-by":"crossref","DOI":"10.1212\/NXI.0000000000001154","article-title":"Neurofilament Light Chain Levels in Multiple Sclerosis Correlate With Lesions Containing Foamy Macrophages and With Acute Axonal Damage","volume":"9","author":"van den Bosch","year":"2022","journal-title":"Neurol. Neuroimmunol. Neuroinflamm."},{"key":"10.1016\/j.cell.2025.06.038_bib133","doi-asserted-by":"crossref","first-page":"2543","DOI":"10.1038\/s41380-024-02524-6","article-title":"Introducing neurofilament light chain measure in psychiatry: current evidence, opportunities, and pitfalls","volume":"29","author":"Bavato","year":"2024","journal-title":"Mol. Psychiatry"},{"key":"10.1016\/j.cell.2025.06.038_bib134","doi-asserted-by":"crossref","first-page":"2508","DOI":"10.1002\/acn3.51234","article-title":"Blood neurofilament light: a critical review of its application to neurologic disease","volume":"7","author":"Barro","year":"2020","journal-title":"Ann. Clin. Transl. Neurol."},{"key":"10.1016\/j.cell.2025.06.038_bib135","doi-asserted-by":"crossref","first-page":"220","DOI":"10.1177\/1352458520912379","article-title":"Neurofilament levels are associated with blood\u2013brain barrier integrity, lymphocyte extravasation, and risk factors following the first demyelinating event in multiple sclerosis","volume":"27","author":"Uher","year":"2021","journal-title":"Mult. Scler."},{"key":"10.1016\/j.cell.2025.06.038_bib136","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1016\/j.neulet.2014.05.044","article-title":"Widespread neuron-specific transgene expression in brain and spinal cord following synapsin promoter-driven AAV9 neonatal intracerebroventricular injection","volume":"576","author":"McLean","year":"2014","journal-title":"Neurosci. Lett."},{"key":"10.1016\/j.cell.2025.06.038_bib137","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1038\/sj.gt.3301905","article-title":"Human synapsin 1 gene promoter confers highly neuron-specific long-term transgene expression from an adenoviral vector in the adult rat brain depending on the transduced area","volume":"10","author":"K\u00fcgler","year":"2003","journal-title":"Gene Ther."},{"key":"10.1016\/j.cell.2025.06.038_bib138","doi-asserted-by":"crossref","first-page":"1272","DOI":"10.1038\/s41551-022-00911-4","article-title":"Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors","volume":"6","author":"Davis","year":"2022","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.cell.2025.06.038_bib139","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1038\/nrneurol.2017.148","article-title":"Antisense oligonucleotides: the next frontier for treatment of neurological disorders","volume":"14","author":"Rinaldi","year":"2018","journal-title":"Nat. Rev. Neurol."},{"key":"10.1016\/j.cell.2025.06.038_bib140","doi-asserted-by":"crossref","first-page":"1526","DOI":"10.1038\/s41587-023-01927-0","article-title":"Precision RNA base editing with engineered and endogenous effectors","volume":"41","author":"Pfeiffer","year":"2023","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib141","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1038\/s41587-021-01133-w","article-title":"Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing","volume":"40","author":"Anzalone","year":"2022","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib142","doi-asserted-by":"crossref","first-page":"500","DOI":"10.1038\/s41587-022-01527-4","article-title":"Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases","volume":"41","author":"Yarnall","year":"2023","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib143","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1038\/s41551-024-01227-1","article-title":"Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing","volume":"9","author":"Pandey","year":"2025","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.cell.2025.06.038_bib144","doi-asserted-by":"crossref","DOI":"10.1126\/science.adt5199","article-title":"Programmable gene insertion in human cells with a laboratory-evolved CRISPR-associated transposase","volume":"388","author":"Witte","year":"2025","journal-title":"Science"},{"key":"10.1016\/j.cell.2025.06.038_bib145","doi-asserted-by":"crossref","first-page":"1151","DOI":"10.1038\/s41587-022-01613-7","article-title":"Predicting prime editing efficiency and product purity by deep learning","volume":"41","author":"Mathis","year":"2023","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib146","first-page":"1","article-title":"Machine learning prediction of prime editing efficiency across diverse chromatin contexts","author":"Mathis","year":"2024","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib147","doi-asserted-by":"crossref","first-page":"2256","DOI":"10.1016\/j.cell.2023.03.034","article-title":"Prediction of efficiencies for diverse prime editing systems in multiple cell types","volume":"186","author":"Yu","year":"2023","journal-title":"Cell"},{"key":"10.1016\/j.cell.2025.06.038_bib148","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1038\/s41587-020-0677-y","article-title":"Predicting the efficiency of prime editing guide RNAs in human cells","volume":"39","author":"Kim","year":"2021","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib149","first-page":"1","article-title":"High-throughput evaluation of genetic variants with prime editing sensor libraries","author":"Gould","year":"2024","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib150","unstructured":"Encoded Therapeutics. (2025). ENDEAVOR: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Infants and Children With SCN1A-Positive Dravet Syndrome. NCT05419492. https:\/\/clinicaltrials.gov\/study\/NCT05419492."},{"key":"10.1016\/j.cell.2025.06.038_bib151","series-title":"Patients With NGLY1 Deficiency. NCT06199531","article-title":"A Phase 1\/2\/3 Open-label, Single Arm, Dose-finding Study to Investigate Long-term Safety, Tolerability and Efficacy of GS-100, an Adeno-associated Virus Serotype 9 (AAV9) Vector-mediated Gene Transfer of Human","year":"2025"},{"key":"10.1016\/j.cell.2025.06.038_bib152","doi-asserted-by":"crossref","first-page":"1220","DOI":"10.1126\/science.adm8386","article-title":"An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery","volume":"384","author":"Huang","year":"2024","journal-title":"Science"},{"key":"10.1016\/j.cell.2025.06.038_bib155","doi-asserted-by":"crossref","first-page":"224","DOI":"10.1038\/s41587-019-0032-3","article-title":"CRISPResso2 provides accurate and rapid genome editing sequence analysis","volume":"37","author":"Clement","year":"2019","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib159","doi-asserted-by":"crossref","first-page":"357","DOI":"10.1038\/nmeth.1923","article-title":"Fast gapped-read alignment with Bowtie 2","volume":"9","author":"Langmead","year":"2012","journal-title":"Nat. Methods"},{"key":"10.1016\/j.cell.2025.06.038_bib160","doi-asserted-by":"crossref","DOI":"10.1093\/gigascience\/giab008","article-title":"Twelve years of SAMtools and BCFtools","volume":"10","author":"Danecek","year":"2021","journal-title":"GigaScience"},{"key":"10.1016\/j.cell.2025.06.038_bib156","doi-asserted-by":"crossref","first-page":"1317","DOI":"10.1038\/s41587-020-0555-7","article-title":"CHANGE-seq reveals genetic and epigenetic effects on CRISPR\u2013Cas9 genome-wide activity","volume":"38","author":"Lazzarotto","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib153","article-title":"Generating Primary Fibroblast Cultures from Mouse Ear and Tail Tissues","volume":"107","author":"Khan","year":"2016","journal-title":"J. Vis. Exp."},{"key":"10.1016\/j.cell.2025.06.038_bib154","doi-asserted-by":"crossref","first-page":"256","DOI":"10.1016\/j.stemcr.2019.12.007","article-title":"Negligible-Cost and Weekend-Free Chemically Defined Human iPSC Culture","volume":"14","author":"Kuo","year":"2020","journal-title":"Stem Cell Rep."},{"key":"10.1016\/j.cell.2025.06.038_bib157","doi-asserted-by":"crossref","DOI":"10.1101\/pdb.top095513","article-title":"Introducing Genes into Mammalian Cells: Viral Vectors","volume":"2020","author":"Sena-Esteves","year":"2020","journal-title":"Cold Spring Harb. Protoc."},{"key":"10.1016\/j.cell.2025.06.038_bib158","doi-asserted-by":"crossref","first-page":"402","DOI":"10.1006\/meth.2001.1262","article-title":"Analysis of Relative Gene Expression Data Using Real-Time Quantitative PCR and the 2-\u0394\u0394CT Method","volume":"25","author":"Livak","year":"2001","journal-title":"Methods"},{"key":"10.1016\/j.cell.2025.06.038_bib161","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1126\/science.1225829","article-title":"A Programmable Dual-RNA\u2013Guided DNA Endonuclease in Adaptive Bacterial Immunity","volume":"337","author":"Jinek","year":"2012","journal-title":"Science"},{"key":"10.1016\/j.cell.2025.06.038_bib162","doi-asserted-by":"crossref","first-page":"1259","DOI":"10.1126\/science.aas9129","article-title":"Engineered CRISPR-Cas9 nuclease with expanded targeting space","volume":"361","author":"Nishimasu","year":"2018","journal-title":"Science"},{"key":"10.1016\/j.cell.2025.06.038_bib163","doi-asserted-by":"crossref","first-page":"471","DOI":"10.1038\/s41587-020-0412-8","article-title":"Continuous evolution of SpCas9 variants compatible with non-G PAMs","volume":"38","author":"Miller","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib164","doi-asserted-by":"crossref","first-page":"101","DOI":"10.1038\/s41589-022-01163-8","article-title":"Engineering a precise adenine base editor with minimal bystander editing","volume":"19","author":"Chen","year":"2023","journal-title":"Nat. Chem. Biol."},{"key":"10.1016\/j.cell.2025.06.038_bib165","doi-asserted-by":"crossref","first-page":"843","DOI":"10.1038\/nbt.4172","article-title":"Improving cytidine and adenine base editors by expression optimization and ancestral reconstruction","volume":"36","author":"Koblan","year":"2018","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib166","doi-asserted-by":"crossref","first-page":"371","DOI":"10.1038\/nbt.3803","article-title":"Increasing the genome-targeting scope and precision of base editing with engineered Cas9-cytidine deaminase fusions","volume":"35","author":"Kim","year":"2017","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib167","doi-asserted-by":"crossref","first-page":"673","DOI":"10.1038\/s41587-022-01533-6","article-title":"Evolution of an adenine base editor into a small, efficient cytosine base editor with low off-target activity","volume":"41","author":"Neugebauer","year":"2023","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.cell.2025.06.038_bib168","article-title":"Phage-assisted evolution of highly active cytosine base editors with enhanced selectivity and minimal sequence context preference","volume":"15","author":"Zhang","year":"2024","journal-title":"Nat. Commun."}],"container-title":["Cell"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0092867425007408?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0092867425007408?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,4,29]],"date-time":"2026-04-29T15:24:13Z","timestamp":1777476253000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0092867425007408"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,8]]},"references-count":168,"journal-issue":{"issue":"16","published-print":{"date-parts":[[2025,8]]}},"alternative-id":["S0092867425007408"],"URL":"https:\/\/doi.org\/10.1016\/j.cell.2025.06.038","relation":{},"ISSN":["0092-8674"],"issn-type":[{"value":"0092-8674","type":"print"}],"subject":[],"published":{"date-parts":[[2025,8]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"In vivo prime editing rescues alternating hemiplegia of childhood in mice","name":"articletitle","label":"Article Title"},{"value":"Cell","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.cell.2025.06.038","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Authors. Published by Elsevier Inc.","name":"copyright","label":"Copyright"}]}}