{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,3]],"date-time":"2026-07-03T23:04:01Z","timestamp":1783119841438,"version":"3.54.6"},"reference-count":76,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Clinical Immunology"],"published-print":{"date-parts":[[2024,1]]},"DOI":"10.1016\/j.clim.2023.109854","type":"journal-article","created":{"date-parts":[[2023,11,25]],"date-time":"2023-11-25T19:33:26Z","timestamp":1700940806000},"page":"109854","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":3,"special_numbering":"C","title":["Challenges for gene editing in common variable immunodeficiency disorders: Current and future prospects"],"prefix":"10.1016","volume":"258","author":[{"given":"Rohan","family":"Ameratunga","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Euphemia","family":"Leung","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"See-Tarn","family":"Woon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edward","family":"Lea","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline","family":"Allan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lydia","family":"Chan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hilary","family":"Longhurst","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Steele","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Russell","family":"Snell","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Klaus","family":"Lehnert","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.clim.2023.109854_bb0005","doi-asserted-by":"crossref","first-page":"17071","DOI":"10.1038\/cddiscovery.2017.71","article-title":"Zinc-finger proteins in health and disease","volume":"3","author":"Cassandri","year":"2017","journal-title":"Cell Death Dis."},{"issue":"1","key":"10.1016\/j.clim.2023.109854_bb0010","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1038\/nrm3486","article-title":"TALENs: a widely applicable technology for targeted genome editing","volume":"14","author":"Joung","year":"2013","journal-title":"Nat. Rev. Mol. Cell Biol."},{"issue":"6096","key":"10.1016\/j.clim.2023.109854_bb0015","doi-asserted-by":"crossref","first-page":"816","DOI":"10.1126\/science.1225829","article-title":"A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity","volume":"337","author":"Jinek","year":"2012","journal-title":"Science."},{"issue":"1","key":"10.1016\/j.clim.2023.109854_bb0020","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1038\/s41392-019-0089-y","article-title":"Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects","volume":"5","author":"Li","year":"2020","journal-title":"Signal Transduct. Target. Ther."},{"issue":"2","key":"10.1016\/j.clim.2023.109854_bb0025","doi-asserted-by":"crossref","first-page":"614","DOI":"10.7150\/thno.47007","article-title":"Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR\/Cas9 genome editing","volume":"11","author":"Zhang","year":"2021","journal-title":"Theranostics."},{"issue":"12","key":"10.1016\/j.clim.2023.109854_bb0030","doi-asserted-by":"crossref","first-page":"2222","DOI":"10.3390\/genes13122222","article-title":"In vivo hematopoietic stem cell genome editing: perspectives and limitations","volume":"13","author":"Psatha","year":"2022","journal-title":"Genes (Basel)"},{"issue":"6","key":"10.1016\/j.clim.2023.109854_bb0035","doi-asserted-by":"crossref","first-page":"722","DOI":"10.1128\/cdli.3.6.722-726.1996","article-title":"Molecular pathology of the X-linked hyper-immunoglobulin M syndrome: detection of wild-type transcripts in a patient with a complex splicing defect of the CD40 ligand","volume":"3","author":"Ameratunga","year":"1996","journal-title":"Clin. Diagn. Lab. Immunol."},{"issue":"9","key":"10.1016\/j.clim.2023.109854_bb0040","doi-asserted-by":"crossref","first-page":"2606","DOI":"10.1016\/j.celrep.2018.04.103","article-title":"Site-specific Gene editing of human hematopoietic stem cells for X-linked hyper-IgM syndrome","volume":"23","author":"Kuo","year":"2018","journal-title":"Cell Rep."},{"issue":"6148","key":"10.1016\/j.clim.2023.109854_bb0045","doi-asserted-by":"crossref","first-page":"1233151","DOI":"10.1126\/science.1233151","article-title":"Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome","volume":"341","author":"Aiuti","year":"2013","journal-title":"Science"},{"issue":"8","key":"10.1016\/j.clim.2023.109854_bb0050","doi-asserted-by":"crossref","first-page":"6708","DOI":"10.1128\/JVI.73.8.6708-6714.1999","article-title":"The resistance of retroviral vectors produced from human cells to serum inactivation in vivo and in vitro is primate species dependent","volume":"73","author":"DePolo","year":"1999","journal-title":"J. Virol."},{"key":"10.1016\/j.clim.2023.109854_bb0055","doi-asserted-by":"crossref","first-page":"203","DOI":"10.1016\/j.omtm.2018.02.009","article-title":"A non-integrating lentiviral approach overcomes Cas9-induced immune rejection to establish an immunocompetent metastatic renal cancer model","volume":"9","author":"Hu","year":"2018","journal-title":"Mol Ther Methods Clin Dev."},{"issue":"3","key":"10.1016\/j.clim.2023.109854_bb0060","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."},{"issue":"13\u201314","key":"10.1016\/j.clim.2023.109854_bb0065","doi-asserted-by":"crossref","first-page":"695","DOI":"10.1089\/hum.2020.182","article-title":"Moving forward after two deaths in a gene therapy trial of Myotubular Myopathy","volume":"31","author":"Wilson","year":"2020","journal-title":"Hum. Gene Ther."},{"issue":"8","key":"10.1016\/j.clim.2023.109854_bb0070","doi-asserted-by":"crossref","first-page":"6729","DOI":"10.1128\/JVI.73.8.6729-6742.1999","article-title":"Variability of human systemic humoral immune responses to adenovirus gene transfer vectors administered to different organs","volume":"73","author":"Harvey","year":"1999","journal-title":"J. Virol."},{"issue":"5","key":"10.1016\/j.clim.2023.109854_bb0075","doi-asserted-by":"crossref","first-page":"969","DOI":"10.3390\/cells10050969","article-title":"CRISPR-Cas and its wide-ranging applications: from human genome editing to environmental implications, technical limitations, Hazards and Bioethical Issues","volume":"10","author":"Piergentili","year":"2021","journal-title":"Cells."},{"issue":"4","key":"10.1016\/j.clim.2023.109854_bb0080","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pbio.3000224","article-title":"Gene-edited babies: what went wrong and what could go wrong","volume":"17","author":"Wang","year":"2019","journal-title":"PLoS Biol."},{"key":"10.1016\/j.clim.2023.109854_bb0085","doi-asserted-by":"crossref","first-page":"1050507","DOI":"10.3389\/fgeed.2022.1050507","article-title":"CRISPR nuclease off-target activity and mitigation strategies","volume":"4","author":"Wienert","year":"2022","journal-title":"Front Genome Ed."},{"issue":"6","key":"10.1016\/j.clim.2023.109854_bb0090","doi-asserted-by":"crossref","first-page":"705","DOI":"10.1007\/s00439-023-02561-1","article-title":"Unintended CRISPR-Cas9 editing outcomes: a review of the detection and prevalence of structural variants generated by gene-editing in human cells","volume":"142","author":"Hunt","year":"2023","journal-title":"Hum. Genet."},{"issue":"6","key":"10.1016\/j.clim.2023.109854_bb0095","doi-asserted-by":"crossref","first-page":"895","DOI":"10.1038\/s41588-021-00838-7","article-title":"Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing","volume":"53","author":"Leibowitz","year":"2021","journal-title":"Nat. Genet."},{"key":"10.1016\/j.clim.2023.109854_bb0100","doi-asserted-by":"crossref","first-page":"936","DOI":"10.1016\/j.omtm.2020.04.015","article-title":"CRISPR-directed therapeutic correction at the NCF1 Locus Is challenged by frequent incidence of chromosomal deletions","volume":"17","author":"Wrona","year":"2020","journal-title":"Mol Ther Methods Clin Dev."},{"issue":"6","key":"10.1016\/j.clim.2023.109854_bb0105","doi-asserted-by":"crossref","first-page":"1044","DOI":"10.1111\/bjh.17269","article-title":"Gene therapy for primary immunodeficiencies","volume":"193","author":"Fox","year":"2021","journal-title":"Br. J. Haematol."},{"key":"10.1016\/j.clim.2023.109854_bb0110","doi-asserted-by":"crossref","DOI":"10.3389\/fimmu.2021.648951","article-title":"Gene therapies for primary immune deficiencies","volume":"12","author":"Kohn","year":"2021","journal-title":"Front. Immunol."},{"issue":"2","key":"10.1016\/j.clim.2023.109854_bb0115","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1016\/j.jaci.2020.05.024","article-title":"Overview of the current status of gene therapy for primary immune deficiencies (PIDs)","volume":"146","author":"Kuo","year":"2020","journal-title":"J. Allergy Clin. Immunol."},{"issue":"5605","key":"10.1016\/j.clim.2023.109854_bb0120","doi-asserted-by":"crossref","first-page":"320","DOI":"10.1126\/science.299.5605.320","article-title":"Second child in French trial is found to have leukemia","volume":"299","author":"Marshall","year":"2003","journal-title":"Science."},{"issue":"5","key":"10.1016\/j.clim.2023.109854_bb0125","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1056\/NEJMoa0805817","article-title":"Gene therapy for immunodeficiency due to adenosine deaminase deficiency","volume":"360","author":"Aiuti","year":"2009","journal-title":"N. Engl. J. Med."},{"issue":"11","key":"10.1016\/j.clim.2023.109854_bb0130","doi-asserted-by":"crossref","first-page":"1026","DOI":"10.1056\/NEJMoa2109329","article-title":"Inhibition of Prekallikrein for hereditary angioedema","volume":"386","author":"Fijen","year":"2022","journal-title":"N. Engl. J. Med."},{"issue":"11","key":"10.1016\/j.clim.2023.109854_bb0135","doi-asserted-by":"crossref","first-page":"1083","DOI":"10.1056\/NEJMe2201729","article-title":"An antisense oligonucleotide for hereditary angioedema","volume":"386","author":"Longhurst","year":"2022","journal-title":"N. Engl. J. Med."},{"issue":"4","key":"10.1016\/j.clim.2023.109854_bb0140","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1097\/MBC.0b013e3283444ddb","article-title":"Severe prekallikrein deficiency due to a homozygous Trp499Stop nonsense mutation","volume":"22","author":"Nakao","year":"2011","journal-title":"Blood Coagul. Fibrinolysis"},{"key":"10.1016\/j.clim.2023.109854_bb0145","doi-asserted-by":"crossref","first-page":"313","DOI":"10.1016\/j.omtm.2018.08.003","article-title":"Disruption of the BCL11A erythroid enhancer reactivates fetal hemoglobin in erythroid cells of patients with \u03b2-Thalassemia major","volume":"10","author":"Psatha","year":"2018","journal-title":"Mol Ther Methods Clin Dev."},{"issue":"13","key":"10.1016\/j.clim.2023.109854_bb0150","doi-asserted-by":"crossref","first-page":"1240","DOI":"10.1056\/NEJMoa1817426","article-title":"CRISPR-edited stem cells in a patient with HIV and Acute Lymphocytic Leukemia","volume":"381","author":"Xu","year":"2019","journal-title":"N. Engl. J. Med."},{"issue":"10","key":"10.1016\/j.clim.2023.109854_bb0155","doi-asserted-by":"crossref","first-page":"901","DOI":"10.1056\/NEJMoa1300662","article-title":"Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV","volume":"370","author":"Tebas","year":"2014","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.clim.2023.109854_bb0160","doi-asserted-by":"crossref","unstructured":"Shen B, Zhang W, Zhang J et al. Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects. Nat. Methods, 11(4), 399\u2013402. doi https:\/\/doi.org\/10.1038\/nmeth.2857. Epub 2014 Mar 1032. (2014).","DOI":"10.1038\/nmeth.2857"},{"issue":"7785","key":"10.1016\/j.clim.2023.109854_bb0165","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"},{"issue":"12","key":"10.1016\/j.clim.2023.109854_bb0170","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."},{"issue":"12","key":"10.1016\/j.clim.2023.109854_bb0175","doi-asserted-by":"crossref","first-page":"839","DOI":"10.1038\/s41573-020-0084-6","article-title":"Base editing: advances and therapeutic opportunities","volume":"19","author":"Porto","year":"2020","journal-title":"Nat. Rev. Drug Discov."},{"issue":"4","key":"10.1016\/j.clim.2023.109854_bb0180","doi-asserted-by":"crossref","first-page":"993","DOI":"10.3390\/cells9040993","article-title":"CRISPR\/Cas9 epigenome editing potential for rare imprinting diseases: a review","volume":"9","author":"Syding","year":"2020","journal-title":"Cells."},{"issue":"9","key":"10.1016\/j.clim.2023.109854_bb0185","doi-asserted-by":"crossref","first-page":"2503","DOI":"10.1016\/j.cell.2021.03.025","article-title":"Genome-wide programmable transcriptional memory by CRISPR-based epigenome editing","volume":"184","author":"Nu\u00f1ez","year":"2021","journal-title":"Cell."},{"key":"10.1016\/j.clim.2023.109854_bb0190","doi-asserted-by":"crossref","DOI":"10.3389\/fimmu.2022.966084","article-title":"Genome editing for primary immunodeficiencies: a therapeutic perspective on Wiskott-Aldrich syndrome","volume":"13","author":"Naseem","year":"2022","journal-title":"Front. Immunol."},{"issue":"5","key":"10.1016\/j.clim.2023.109854_bb0195","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1038\/s41568-022-00441-w","article-title":"CRISPR in cancer biology and therapy","volume":"22","author":"Katti","year":"2022","journal-title":"Nat. Rev. Cancer"},{"key":"10.1016\/j.clim.2023.109854_bb0200","doi-asserted-by":"crossref","first-page":"2130","DOI":"10.3389\/fimmu.2018.02130","article-title":"Assessing disease severity in common variable immunodeficiency disorders (CVID) and CVID-like disorders","volume":"9","author":"Ameratunga","year":"2018","journal-title":"Front. Immunol."},{"issue":"7","key":"10.1016\/j.clim.2023.109854_bb0205","doi-asserted-by":"crossref","first-page":"549","DOI":"10.1080\/1744666X.2018.1481750","article-title":"Keeping it in the family: the case for considering late-onset combined immunodeficiency a subset of common variable immunodeficiency disorders","volume":"14","author":"Ameratunga","year":"2018","journal-title":"Expert. Rev. Clin. Immunol."},{"issue":"doi","key":"10.1016\/j.clim.2023.109854_bb0210","doi-asserted-by":"crossref","first-page":"694","DOI":"10.3389\/fimmu.2018.00694","article-title":"Delayed diagnosis and complications of predominantly antibody deficiencies in a cohort of Australian adults","volume":"9","author":"Slade","year":"2018","journal-title":"Front. Immunol."},{"issue":"3","key":"10.1016\/j.clim.2023.109854_bb0215","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1111\/cei.13595","article-title":"Bronchiectasis is associated with delayed diagnosis and adverse outcomes in the New Zealand common variable immunodeficiency disorders cohort study","volume":"204","author":"Ameratunga","year":"2021","journal-title":"Clin. Exp. Immunol."},{"key":"10.1016\/j.clim.2023.109854_bb0220","doi-asserted-by":"crossref","first-page":"706796","DOI":"10.3389\/fimmu.2021.706796","article-title":"Are all primary immunodeficiency disorders inborn errors of immunity?","volume":"12","author":"Ameratunga","year":"2021","journal-title":"Front. Immunol."},{"key":"10.1016\/j.clim.2023.109854_bb0225","doi-asserted-by":"crossref","first-page":"176","DOI":"10.1016\/j.coi.2021.05.010","article-title":"There is no gene for CVID - novel monogenetic causes for primary antibody deficiency","volume":"72","author":"Ramirez","year":"2021","journal-title":"Curr. Opin. Immunol."},{"issue":"2","key":"10.1016\/j.clim.2023.109854_bb0230","doi-asserted-by":"crossref","first-page":"203","DOI":"10.1111\/cei.12178","article-title":"New diagnostic criteria for common variable immune deficiency (CVID), which may assist with decisions to treat with intravenous or subcutaneous immunoglobulin","volume":"174","author":"Ameratunga","year":"2013","journal-title":"Clin. Exp. Immunol."},{"issue":"1","key":"10.1016\/j.clim.2023.109854_bb0235","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1016\/j.jaip.2015.07.025","article-title":"International consensus document (ICON): common variable immunodeficiency disorders","volume":"4","author":"Bonilla","year":"2016","journal-title":"J Allergy Clin Immunol Pract"},{"issue":"6","key":"10.1016\/j.clim.2023.109854_bb0240","doi-asserted-by":"crossref","first-page":"1763","DOI":"10.1016\/j.jaip.2019.02.004","article-title":"The European Society for Immunodeficiencies (ESID) registry working definitions for the clinical diagnosis of inborn errors of immunity","volume":"7","author":"Seidel","year":"2019","journal-title":"J Allergy Clin Immunol Pract"},{"issue":"3","key":"10.1016\/j.clim.2023.109854_bb0245","doi-asserted-by":"crossref","first-page":"257","DOI":"10.1586\/1744666X.2016.1126509","article-title":"Application of diagnostic and treatment criteria for common variable immunodeficiency disorder","volume":"12","author":"Ameratunga","year":"2016","journal-title":"Expert. Rev. Clin. Immunol."},{"key":"10.1016\/j.clim.2023.109854_bb0250","doi-asserted-by":"crossref","first-page":"1190","DOI":"10.3389\/fimmu.2017.01190","article-title":"Unexpectedly high prevalence of common variable immunodeficiency in Finland","volume":"8","author":"Selenius","year":"2017","journal-title":"Front. Immunol."},{"issue":"10","key":"10.1016\/j.clim.2023.109854_bb0255","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0140473","article-title":"The incidence and prevalence of common variable immunodeficiency disease in Taiwan. A population-based study","volume":"10","author":"Tseng","year":"2015","journal-title":"PLoS One"},{"key":"10.1016\/j.clim.2023.109854_bb0260","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1146\/annurev-pathmechdis-031521-024229","article-title":"Common variable immunodeficiency: more pathways than roads to Rome","volume":"18","author":"Peng","year":"2023","journal-title":"Annu. Rev. Pathol."},{"issue":"23","key":"10.1016\/j.clim.2023.109854_bb0265","first-page":"00174","article-title":"The rapidly expanding genetic spectrum of common variable immunodeficiency-like disorders","volume":"14","author":"Ameratunga","year":"2023","journal-title":"J Allergy Clin Immunol Pract"},{"issue":"8","key":"10.1016\/j.clim.2023.109854_bb0270","doi-asserted-by":"crossref","first-page":"820","DOI":"10.1038\/ng1600","article-title":"Mutations in TNFRSF13B encoding TACI are associated with common variable immunodeficiency in humans","volume":"37","author":"Salzer","year":"2005","journal-title":"Nat. Genet."},{"issue":"17","key":"10.1016\/j.clim.2023.109854_bb0275","doi-asserted-by":"crossref","first-page":"7193","DOI":"10.1073\/pnas.0700815104","article-title":"Role for Msh5 in the regulation of Ig class switch recombination","volume":"104","author":"Sekine","year":"2007","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"issue":"3","key":"10.1016\/j.clim.2023.109854_bb0280","doi-asserted-by":"crossref","first-page":"389","DOI":"10.1016\/j.ajhg.2015.07.008","article-title":"Haploinsufficiency of the NF-kappaB1 subunit p50 in common variable immunodeficiency","volume":"97","author":"Fliegauf","year":"2015","journal-title":"Am. J. Hum. Genet."},{"issue":"5","key":"10.1016\/j.clim.2023.109854_bb0285","doi-asserted-by":"crossref","first-page":"812","DOI":"10.1016\/j.ajhg.2013.09.009","article-title":"Germline mutations in NFKB2 implicate the noncanonical NF-kappaB pathway in the pathogenesis of common variable immunodeficiency","volume":"93","author":"Chen","year":"2013","journal-title":"Am. J. Hum. Genet."},{"issue":"1","key":"10.1016\/j.clim.2023.109854_bb0290","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1007\/s12016-019-08765-6","article-title":"Perspective: evolving concepts in the diagnosis and understanding of common variable immunodeficiency disorders (CVID)","volume":"59","author":"Ameratunga","year":"2020","journal-title":"Clin. Rev. Allergy Immunol."},{"issue":"1","key":"10.1016\/j.clim.2023.109854_bb0295","doi-asserted-by":"crossref","first-page":"243","DOI":"10.1038\/s41436-018-0012-x","article-title":"Clinical implications of systematic phenotyping and exome sequencing in patients with primary antibody deficiency","volume":"21","author":"Abolhassani","year":"2019","journal-title":"Genet. Med."},{"issue":"9","key":"10.1016\/j.clim.2023.109854_bb0300","doi-asserted-by":"crossref","first-page":"656","DOI":"10.1182\/blood.2019000929","article-title":"Current genetic landscape in common variable immune deficiency","volume":"135","author":"Abolhassani","year":"2020","journal-title":"Blood"},{"issue":"3","key":"10.1016\/j.clim.2023.109854_bb0305","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1016\/j.iac.2020.03.001","article-title":"Defining common variable immunodeficiency disorders in 2020","volume":"40","author":"Ameratunga","year":"2020","journal-title":"Immunol. Allergy Clin. N. Am."},{"issue":"10","key":"10.1016\/j.clim.2023.109854_bb0310","doi-asserted-by":"crossref","DOI":"10.1038\/cti.2017.41","article-title":"Epistatic interactions between mutations of TACI (TNFRSF13B) and TCF3 result in a severe primary immunodeficiency disorder and systemic lupus erythematosus","volume":"6","author":"Ameratunga","year":"2017","journal-title":"Clin. Transl. Immunol."},{"issue":"Gen Rep","key":"10.1016\/j.clim.2023.109854_bb0315","first-page":"22","article-title":"Discussion on the influence of heredity on disease, with special reference to tuberculosis, Cancer, and diseases of the nervous system: introductory address","volume":"2","author":"Bateson.","year":"1909","journal-title":"Proc. R. Soc. Med."},{"issue":"1","key":"10.1016\/j.clim.2023.109854_bb0320","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1007\/s10875-012-9793-x","article-title":"Clinical variability of family members with the C104R mutation in transmembrane activator and calcium modulator and cyclophilin ligand interactor (TACI)","volume":"33","author":"Koopmans","year":"2013","journal-title":"J. Clin. Immunol."},{"issue":"2","key":"10.1016\/j.clim.2023.109854_bb0325","doi-asserted-by":"crossref","first-page":"261","DOI":"10.1007\/s12016-017-8645-0","article-title":"Review: diagnosing common variable immunodeficiency disorder in the era of genome sequencing","volume":"54","author":"Ameratunga","year":"2018","journal-title":"Clin. Rev. Allergy Immunol."},{"key":"10.1016\/j.clim.2023.109854_bb0330","doi-asserted-by":"crossref","first-page":"1965","DOI":"10.3389\/fimmu.2017.01965","article-title":"Clinical implications of Digenic inheritiance and epistasis in primary immunodeficiency disorders","volume":"8","author":"Ameratunga","year":"2018","journal-title":"Front. Immunol."},{"key":"10.1016\/j.clim.2023.109854_bb0335","doi-asserted-by":"crossref","first-page":"7335","DOI":"10.1038\/ncomms8335","article-title":"Monozygotic twins discordant for common variable immunodeficiency reveal impaired DNA demethylation during naive-to-memory B-cell transition","volume":"6","author":"Rodriguez-Cortez","year":"2015","journal-title":"Nat. Commun."},{"issue":"1","key":"10.1016\/j.clim.2023.109854_bb0340","doi-asserted-by":"crossref","first-page":"1779","DOI":"10.1038\/s41467-022-29450-x","article-title":"Single-cell atlas of common variable immunodeficiency shows germinal center-associated epigenetic dysregulation in B-cell responses","volume":"13","author":"Rodr\u00edguez-Ubreva","year":"2022","journal-title":"Nat. Commun."},{"key":"10.1016\/j.clim.2023.109854_bb0345","doi-asserted-by":"crossref","first-page":"2678","DOI":"10.3389\/fimmu.2019.02678","article-title":"All patients with common variable immunodeficiency disorders (CVID) should be routinely offered diagnostic genetic testing","volume":"10","author":"Ameratunga","year":"2019","journal-title":"Front. Immunol."},{"issue":"2","key":"10.1016\/j.clim.2023.109854_bb0350","doi-asserted-by":"crossref","first-page":"226","DOI":"10.1007\/s12016-020-08828-z","article-title":"Perspective: application of the American College of Medical Genetics Variant Interpretation Criteria to common variable immunodeficiency disorders","volume":"61","author":"Ameratunga","year":"2021","journal-title":"Clin. Rev. Allergy Immunol."},{"issue":"5","key":"10.1016\/j.clim.2023.109854_bb0355","doi-asserted-by":"crossref","first-page":"405","DOI":"10.1038\/gim.2015.30","article-title":"Standards and guidelines for the interpretation of sequence variants: a joint consensus recommendation of the American College of Medical Genetics and Genomics and the Association for Molecular Pathology","volume":"17","author":"Richards","year":"2015","journal-title":"Genet. Med."},{"issue":"10","key":"10.1016\/j.clim.2023.109854_bb0360","first-page":"022","article-title":"CVID-associated B cell activating factor receptor variants change receptor oligomerization, ligand binding, and signaling responses","volume":"29","author":"Block","year":"2022","journal-title":"J. Clin. Immunol."},{"key":"10.1016\/j.clim.2023.109854_bb0365","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1111\/j.1749-6632.2011.06238.x","article-title":"Primary immune deficiency disorders in the South Pacific: the clinical utility of a customized genetic testing program in New Zealand","volume":"1238","author":"Ameratunga","year":"2011","journal-title":"Ann. N. Y. Acad. Sci."},{"issue":"1","key":"10.1016\/j.clim.2023.109854_bb0370","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1186\/1710-1492-6-12","article-title":"The clinical utility of molecular diagnostic testing for primary immune deficiency disorders: a case based review","volume":"6","author":"Ameratunga","year":"2010","journal-title":"Allergy, Asthma Clin. Immunol."},{"issue":"1304","key":"10.1016\/j.clim.2023.109854_bb0375","first-page":"46","article-title":"Customised molecular diagnosis of primary immune deficiency disorders in New Zealand: an efficient strategy for a small developed country","volume":"122","author":"Ameratunga","year":"2009","journal-title":"N. Z. Med. J."},{"issue":"6","key":"10.1016\/j.clim.2023.109854_bb0380","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1056\/NEJMoa2107454","article-title":"CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis","volume":"385","author":"Gillmore","year":"2021","journal-title":"N. Engl. J. Med."}],"container-title":["Clinical Immunology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1521661623006186?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1521661623006186?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,26]],"date-time":"2025-10-26T16:11:12Z","timestamp":1761495072000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1521661623006186"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,1]]},"references-count":76,"alternative-id":["S1521661623006186"],"URL":"https:\/\/doi.org\/10.1016\/j.clim.2023.109854","relation":{},"ISSN":["1521-6616"],"issn-type":[{"value":"1521-6616","type":"print"}],"subject":[],"published":{"date-parts":[[2024,1]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Challenges for gene editing in common variable immunodeficiency disorders: Current and future prospects","name":"articletitle","label":"Article Title"},{"value":"Clinical Immunology","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.clim.2023.109854","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 Elsevier Inc. All rights reserved.","name":"copyright","label":"Copyright"}],"article-number":"109854"}}