{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T14:56:10Z","timestamp":1789052170772,"version":"build-2803163510"},"reference-count":194,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2022,3,17]],"date-time":"2022-03-17T00:00:00Z","timestamp":1647475200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100003554","name":"Lundbeckfonden","doi-asserted-by":"publisher","award":["R238-2016-534 3349"],"award-info":[{"award-number":["R238-2016-534 3349"]}],"id":[{"id":"10.13039\/501100003554","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100011958","name":"Danmarks Frie Forskningsfond","doi-asserted-by":"publisher","award":["0134-00113B 0242-00009B 9144-00001B"],"award-info":[{"award-number":["0134-00113B 0242-00009B 9144-00001B"]}],"id":[{"id":"10.13039\/501100011958","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100009388","name":"Aarhus Institute of Advanced Studies, Aarhus Universitet","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100009388","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100011264","name":"FP7 People: Marie-Curie Actions","doi-asserted-by":"publisher","award":["609033"],"award-info":[{"award-number":["609033"]}],"id":[{"id":"10.13039\/100011264","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100009708","name":"Novo Nordisk Fonden","doi-asserted-by":"publisher","award":["NNF19OC0058238 NNF17OC0028894"],"award-info":[{"award-number":["NNF19OC0058238 NNF17OC0028894"]}],"id":[{"id":"10.13039\/501100009708","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100012774","name":"Innovationsfonden","doi-asserted-by":"publisher","award":["8056-00010B"],"award-info":[{"award-number":["8056-00010B"]}],"id":[{"id":"10.13039\/100012774","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100002808","name":"Carlsbergfondet","doi-asserted-by":"publisher","award":["CF20-0424 CF17-0129"],"award-info":[{"award-number":["CF20-0424 CF17-0129"]}],"id":[{"id":"10.13039\/501100002808","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100006309","name":"Riisfort Fonden","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100006309","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Genome Ed."],"abstract":"<jats:p>Defects in the DOCK8 gene causes combined immunodeficiency termed DOCK8 immunodeficiency syndrome (DIDS). DIDS previously belonged to the disease category of autosomal recessive hyper IgE syndrome (AR-HIES) but is now classified as a combined immunodeficiency (CID). This genetic disorder induces early onset of susceptibility to severe recurrent viral and bacterial infections, atopic diseases and malignancy resulting in high morbidity and mortality. This pathological state arises from impairment of actin polymerization and cytoskeletal rearrangement, which induces improper immune cell migration-, survival-, and effector functions. Owing to the severity of the disease, early allogenic hematopoietic stem cell transplantation is recommended even though it is associated with risk of unintended adverse effects, the need for compatible donors, and high expenses. So far, no alternative therapies have been developed, but the monogenic recessive nature of the disease suggests that gene therapy may be applied. The advent of the CRISPR\/Cas gene editing system heralds a new era of possibilities in precision gene therapy, and positive results from clinical trials have already suggested that the tool may provide definitive cures for several genetic disorders. Here, we discuss the potential application of different CRISPR\/Cas-mediated genetic therapies to correct the DOCK8 gene. Our findings encourage the pursuit of CRISPR\/Cas-based gene editing approaches, which may constitute more precise, affordable, and low-risk definitive treatment options for DOCK8 deficiency.<\/jats:p>","DOI":"10.3389\/fgeed.2022.793010","type":"journal-article","created":{"date-parts":[[2022,3,17]],"date-time":"2022-03-17T09:02:11Z","timestamp":1647507731000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":10,"title":["CRISPR\/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency Syndrome"],"prefix":"10.3389","volume":"4","author":[{"given":"Sujan","family":"Ravendran","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabina S\u00e1nchez","family":"Hern\u00e1ndez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Saskia","family":"K\u00f6nig","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rasmus O.","family":"Bak","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2022,3,17]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1038\/ncomms13173","article-title":"Semi-Automated Closed System Manufacturing of Lentivirus Gene-Modified Haematopoietic Stem Cells for Gene Therapy","volume":"7","author":"Adair","year":"2016","journal-title":"Nat. Commun."},{"key":"B2","doi-asserted-by":"publisher","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"},{"key":"B3","doi-asserted-by":"publisher","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."},{"key":"B4","doi-asserted-by":"publisher","first-page":"2410","DOI":"10.1126\/science.1070104","article-title":"Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning","volume":"296","author":"Aiuti","year":"2002","journal-title":"Science"},{"key":"B5","doi-asserted-by":"publisher","first-page":"852","DOI":"10.1016\/j.jaci.2016.02.022","article-title":"Hematopoietic Stem Cell Transplantation Outcomes for 11 Patients with Dedicator of Cytokinesis 8 Deficiency","volume":"138","author":"Al-Herz","year":"2016","journal-title":"J. Allergy Clin. Immunol."},{"key":"B6","doi-asserted-by":"publisher","first-page":"104","DOI":"10.1016\/j.clim.2014.04.005","article-title":"Successful Interferon-Alpha 2b Therapy for Unremitting Warts in a Patient with DOCK8 Deficiency","volume":"153","author":"Al-Zahrani","year":"2014","journal-title":"Clin. Immunol."},{"key":"B7","doi-asserted-by":"publisher","first-page":"55","DOI":"10.1007\/s10875-012-9769-x","article-title":"Clinical, Immunological and Molecular Characterization of DOCK8 and DOCK8-like Deficient Patients: Single center Experience of Twenty Five Patients","volume":"33","author":"Alsum","year":"2013","journal-title":"J. Clin. Immunol."},{"key":"B8","doi-asserted-by":"publisher","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":"B9","doi-asserted-by":"publisher","first-page":"108","DOI":"10.1186\/1477-7827-12-108","article-title":"International Regulatory Landscape and Integration of Corrective Genome Editing into In Vitro Fertilization","volume":"12","author":"Araki","year":"2014","journal-title":"Reprod. Biol. Endocrinol."},{"key":"B10","doi-asserted-by":"publisher","first-page":"848","DOI":"10.1016\/j.jaip.2018.10.035","article-title":"Hematopoietic Stem Cell Transplantation as Treatment for Patients with DOCK8 Deficiency","volume":"7","author":"Aydin","year":"2019","journal-title":"J. Allergy Clin. Immunol. Pract."},{"key":"B11","doi-asserted-by":"publisher","first-page":"189","DOI":"10.1007\/s10875-014-0126-0","article-title":"DOCK8 Deficiency: Clinical and Immunological Phenotype and Treatment Options - a Review of 136 Patients","volume":"35","author":"Aydin","year":"2015","journal-title":"J. Clin. Immunol."},{"key":"B12","doi-asserted-by":"publisher","first-page":"358","DOI":"10.1038\/nprot.2017.143","article-title":"CRISPR\/Cas9 Genome Editing in Human Hematopoietic Stem Cells","volume":"13","author":"Bak","year":"2018","journal-title":"Nat. Protoc."},{"key":"B13","doi-asserted-by":"publisher","first-page":"1","DOI":"10.7554\/eLife.27873","article-title":"Multiplexed Genetic Engineering of Human Hematopoietic Stem and Progenitor Cells Using CRISPR\/Cas9 and AAV6","volume":"6","author":"Bak","year":"2017","journal-title":"Elife"},{"key":"B14","doi-asserted-by":"publisher","first-page":"600","DOI":"10.1016\/j.tig.2018.05.004","article-title":"Gene Editing on Center Stage","volume":"34","author":"Bak","year":"2018","journal-title":"Trends Genet."},{"key":"B15","doi-asserted-by":"publisher","first-page":"750","DOI":"10.1016\/j.celrep.2017.06.064","article-title":"CRISPR-mediated Integration of Large Gene Cassettes Using AAV Donor Vectors","volume":"20","author":"Bak","year":"2017","journal-title":"Cel Rep."},{"key":"B16","doi-asserted-by":"publisher","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":"Cgt"},{"key":"B17","doi-asserted-by":"publisher","first-page":"187","DOI":"10.3747\/co.26.5033","article-title":"A General Practitioner's Guide to Hematopoietic Stem-Cell Transplantation","volume":"26","author":"Bazinet","year":"2019","journal-title":"Curr. Oncol."},{"key":"B18","doi-asserted-by":"publisher","first-page":"358","DOI":"10.1038\/emm.2004.47","article-title":"The Role of Rho GTPases in the Regulation of the Rearrangement of Actin Cytoskeleton and Cell Movement","volume":"36","author":"Begum","year":"2004","journal-title":"Exp. Mol. Med."},{"key":"B19","doi-asserted-by":"publisher","first-page":"75","DOI":"10.1016\/j.clim.2017.06.003","article-title":"DOCK8 Deficiency: Insights into Pathophysiology, Clinical Features and Management","volume":"181","author":"Biggs","year":"2017","journal-title":"Clin. Immunol."},{"key":"B20","doi-asserted-by":"publisher","first-page":"37","DOI":"10.1111\/imr.12737","article-title":"T Cell Interactions with B Cells during Germinal center Formation, a Three\u2010step Model","volume":"288","author":"Biram","year":"2019","journal-title":"Immunol. Rev."},{"key":"B21","doi-asserted-by":"publisher","first-page":"21","DOI":"10.1016\/j.cels.2016.12.010","article-title":"Lessons from Enzyme Kinetics Reveal Specificity Principles for RNA-Guided Nucleases in RNA Interference and CRISPR-Based Genome Editing","volume":"4","author":"Bisaria","year":"2017","journal-title":"Cel Syst."},{"key":"B22","doi-asserted-by":"publisher","first-page":"1611","DOI":"10.1016\/j.ymthe.2020.12.010","article-title":"Genome Editing in Human Hematopoietic Stem and Progenitor Cells via CRISPR-Cas9-Mediated Homology-independent Targeted Integration","volume":"29","author":"Bloomer","year":"2021","journal-title":"Mol. Ther."},{"key":"B23","doi-asserted-by":"publisher","first-page":"743","DOI":"10.1146\/annurev.biochem.72.121801.161742","article-title":"Biology of the P21-Activated Kinases","volume":"72","author":"Bokoch","year":"2003","journal-title":"Annu. Rev. Biochem."},{"key":"B24","doi-asserted-by":"publisher","first-page":"317","DOI":"10.1016\/j.molmed.2016.02.002","article-title":"Treating Immunodeficiency through HSC Gene Therapy","volume":"22","author":"Booth","year":"2016","journal-title":"Trends Mol. Med."},{"key":"B25","doi-asserted-by":"publisher","first-page":"R15","DOI":"10.1093\/hmg\/ddz170","article-title":"Gene Therapy for Primary Immunodeficiency","volume":"28","author":"Booth","year":"2019","journal-title":"Hum. Mol. Genet."},{"key":"B26","doi-asserted-by":"publisher","first-page":"66","DOI":"10.1007\/s10875-020-00758-x","article-title":"Human Inborn Errors of Immunity: 2019 Update of the IUIS Phenotypical Classification","volume":"40","author":"Bousfiha","year":"2020","journal-title":"J. Clin. Immunol."},{"key":"B27","doi-asserted-by":"publisher","first-page":"1918","DOI":"10.1056\/NEJMoa1003548","article-title":"Stem-Cell Gene Therapy for the Wiskott-Aldrich Syndrome","volume":"363","author":"Boztug","year":"2010","journal-title":"N. Engl. J. Med."},{"key":"B28","first-page":"366","article-title":"The Cost of Hematopoietic Stem-Cell Transplantation in the United States","volume":"10","author":"Broder","year":"2017","journal-title":"Am. Health Drug Benefits"},{"key":"B29","doi-asserted-by":"publisher","first-page":"651","DOI":"10.1007\/s12026-016-8883-x","article-title":"The Clinical and Laboratory Spectrum of Dedicator of Cytokinesis 8 Immunodeficiency Syndrome in Patients with a Unique Mutation","volume":"65","author":"Broides","year":"2017","journal-title":"Immunol. Res."},{"key":"B30","doi-asserted-by":"publisher","first-page":"1253","DOI":"10.1038\/3233","article-title":"Thymic Lymphoproliferative Disease after Successful Correction of CD40 Ligand Deficiency by Gene Transfer in Mice","volume":"4","author":"Brown","year":"1998","journal-title":"Nat. Med."},{"key":"B31","doi-asserted-by":"publisher","first-page":"53","DOI":"10.1084\/jem.20141143","article-title":"Cdc42 Is a Key Regulator of B Cell Differentiation and Is Required for Antiviral Humoral Immunity","volume":"212","author":"Burbage","year":"2015","journal-title":"J. Exp. Med."},{"key":"B32","doi-asserted-by":"publisher","first-page":"2083","DOI":"10.1172\/JCI32949","article-title":"Retroviral Integration and Human Gene Therapy","volume":"117","author":"Bushman","year":"2007","journal-title":"J. Clin. Invest."},{"key":"B33","doi-asserted-by":"publisher","first-page":"164","DOI":"10.1016\/j.clim.2014.03.008","article-title":"Expansion of CCR4+ Activated T Cells Is Associated with Memory B Cell Reduction in DOCK8-Deficient Patients","volume":"152","author":"Caracciolo","year":"2014","journal-title":"Clin. Immunol."},{"key":"B34","doi-asserted-by":"publisher","first-page":"1665","DOI":"10.3390\/cells9071665","article-title":"DNA Damage: From Threat to Treatment","volume":"9","author":"Carusillo","year":"2020","journal-title":"Cells"},{"key":"B35","doi-asserted-by":"publisher","first-page":"318","DOI":"10.1038\/nature09328","article-title":"Transfusion independence and HMGA2 Activation after Gene Therapy of Human \u03b2-thalassaemia","volume":"467","author":"Cavazzana-Calvo","year":"2010","journal-title":"Nature"},{"key":"B36","doi-asserted-by":"publisher","first-page":"1580","DOI":"10.1016\/j.jval.2020.06.020","article-title":"Access and Unmet Needs of Orphan Drugs in 194 Countries and 6 Areas: A Global Policy Review with Content Analysis","volume":"23","author":"Chan","year":"2020","journal-title":"Value in Health"},{"key":"B37","doi-asserted-by":"publisher","first-page":"1133","DOI":"10.1038\/s41467-018-03475-7","article-title":"CtIP Fusion to Cas9 Enhances Transgene Integration by Homology-dependent Repair","volume":"9","author":"Charpentier","year":"2018","journal-title":"Nat. Commun."},{"key":"B38","doi-asserted-by":"publisher","first-page":"2159","DOI":"10.4049\/jimmunol.1200156","article-title":"Cytokine Secretion by CD4+ T Cells at the Immunological Synapse Requires Cdc42-dependent Local Actin Remodeling but Not Microtubule Organizing center Polarity","volume":"189","author":"Chemin","year":"2012","journal-title":"J.I."},{"key":"B39","doi-asserted-by":"publisher","first-page":"757","DOI":"10.1534\/genetics.110.120717","article-title":"Targeting DNA Double-Strand Breaks with TAL Effector Nucleases","volume":"186","author":"Christian","year":"2010","journal-title":"Genetics"},{"key":"B40","doi-asserted-by":"publisher","first-page":"79","DOI":"10.1001\/archdermatol.2011.262","article-title":"Cutaneous Manifestations of DOCK8 Deficiency Syndrome","volume":"148","author":"Chu","year":"2012","journal-title":"Arch. Dermatol."},{"key":"B41","doi-asserted-by":"publisher","first-page":"543","DOI":"10.1038\/nbt.3198","article-title":"Increasing the Efficiency of Homology-Directed Repair for CRISPR-Cas9-Induced Precise Gene Editing in Mammalian Cells","volume":"33","author":"Chu","year":"2015","journal-title":"Nat. Biotechnol."},{"key":"B42","doi-asserted-by":"publisher","first-page":"45","DOI":"10.1182\/blood-2016-01-688226","article-title":"Update on the Safety and Efficacy of Retroviral Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency","volume":"128","author":"Cicalese","year":"2016","journal-title":"Blood"},{"key":"B43","doi-asserted-by":"publisher","first-page":"819","DOI":"10.1126\/science.1231143","article-title":"Multiplex Genome Engineering Using CRISPR\/Cas Systems","volume":"339","author":"Cong","year":"2013","journal-title":"Science"},{"key":"B44","doi-asserted-by":"publisher","first-page":"1813","DOI":"10.1056\/nejmra052638","article-title":"Hematopoietic Stem-Cell Transplantation","volume":"354","author":"Copelan","year":"2006","journal-title":"N. Engl. J. Med."},{"key":"B45","doi-asserted-by":"publisher","first-page":"797","DOI":"10.1038\/ncb1280","article-title":"A Novel and Evolutionarily Conserved PtdIns(3,4,5)P3-Binding Domain Is Necessary for DOCK180 Signalling","volume":"7","author":"C\u00f4t\u00e9","year":"2005","journal-title":"Nat. Cel Biol."},{"key":"B46","doi-asserted-by":"publisher","first-page":"383","DOI":"10.1016\/j.tcb.2007.05.001","article-title":"GEF what? Dock180 and Related Proteins Help Rac to Polarize Cells in New Ways","volume":"17","author":"C\u00f4t\u00e9","year":"2007","journal-title":"Trends Cel Biol."},{"key":"B47","doi-asserted-by":"publisher","first-page":"4901","DOI":"10.1242\/jcs.00219","article-title":"Identification of an Evolutionarily Conserved Superfamily of DOCK180-Related Proteins with Guanine Nucleotide Exchange Activity","volume":"115","author":"Co\u0302te\u0301","year":"2002","journal-title":"J. Cel Sci."},{"key":"B48","doi-asserted-by":"publisher","first-page":"2052","DOI":"10.1182\/blood-2013-02-482331","article-title":"DOCK8 Is Critical for the Survival and Function of NKT Cells","volume":"122","author":"Crawford","year":"2013","journal-title":"Blood"},{"key":"B49","doi-asserted-by":"publisher","first-page":"677","DOI":"10.1038\/s41591-021-01284-y","article-title":"Gene Replacement of \u03b1-globin with \u03b2-globin Restores Hemoglobin Balance in \u03b2-thalassemia-derived Hematopoietic Stem and Progenitor Cells","volume":"27","author":"Cromer","year":"2021","journal-title":"Nat. Med."},{"key":"B50","doi-asserted-by":"publisher","first-page":"2431","DOI":"10.1016\/j.ymthe.2018.06.002","article-title":"Global Transcriptional Response to CRISPR\/Cas9-AAV6-Based Genome Editing in CD34+ Hematopoietic Stem and Progenitor Cells","volume":"26","author":"Cromer","year":"2018","journal-title":"Mol. Ther."},{"key":"B51","doi-asserted-by":"publisher","first-page":"479","DOI":"10.1038\/nature.2016.20988","article-title":"CRISPR Gene-Editing Tested in a Person for the First Time","volume":"539","author":"Cyranoski","year":"2016","journal-title":"Nature"},{"key":"B52","doi-asserted-by":"publisher","first-page":"D8","DOI":"10.1093\/nar\/gkx1095","article-title":"Database Resources of the National Center for Biotechnology Information","volume":"46","year":"2018","journal-title":"Nucleic Acids Res."},{"key":"B53","doi-asserted-by":"publisher","first-page":"2598","DOI":"10.1182\/blood.2020008503","article-title":"Enhanced Homology-Directed Repair for Highly Efficient Gene Editing in Hematopoietic Stem\/Progenitor Cells","volume":"137","author":"De Ravin","year":"2021","journal-title":"Blood"},{"key":"B54","doi-asserted-by":"publisher","first-page":"9","DOI":"10.1126\/scitranslmed.aah3480","article-title":"CRISPR-Cas9 Gene Repair of Hematopoietic Stem Cells from Patients with X-Linked Chronic Granulomatous Disease","volume":"9","author":"De Ravin","year":"2017","journal-title":"Sci. Transl. Med."},{"key":"B55","doi-asserted-by":"publisher","first-page":"424","DOI":"10.1038\/nbt.3513","article-title":"Targeted Gene Addition in Human CD34+ Hematopoietic Cells for Correction of X-Linked Chronic Granulomatous Disease","volume":"34","author":"De Ravin","year":"2016","journal-title":"Nat. Biotechnol."},{"key":"B56","doi-asserted-by":"publisher","first-page":"1889841","DOI":"10.1080\/20016689.2021.1889841","article-title":"An Updated Cost-Utility Model for Onasemnogene Abeparvovec (Zolgensma) in Spinal Muscular Atrophy Type 1 Patients and Comparison with Evaluation by the Institute for Clinical and Effectiveness Review (ICER)","volume":"9","author":"Dean","year":"2021","journal-title":"J. Market Access Health Pol."},{"key":"B57","doi-asserted-by":"publisher","first-page":"384","DOI":"10.1038\/nature20134","article-title":"CRISPR\/Cas9 \u03b2-globin Gene Targeting in Human Haematopoietic Stem Cells","volume":"539","author":"Dever","year":"2016","journal-title":"Nature"},{"key":"B58","doi-asserted-by":"publisher","first-page":"1450","DOI":"10.1093\/annonc\/mdm064","article-title":"Hematopoietic Cell Transplantation: Progress and Obstacles","volume":"18","author":"Devetten","year":"2007","journal-title":"Ann. Oncol."},{"key":"B59","doi-asserted-by":"publisher","first-page":"360ra134","DOI":"10.1126\/scitranslmed.aaf9336","article-title":"Selection-free Genome Editing of the Sickle Mutation in Human Adult Hematopoietic Stem\/progenitor Cells","volume":"8","author":"DeWitt","year":"2016","journal-title":"Sci. Transl. Med."},{"key":"B60","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/978-981-15-6407-9_1","article-title":"Regulatory T Cells: Concept, Classification, Phenotype, and Biological Characteristics","volume-title":"T Regulatory Cells in Human Health and Diseases","author":"Du","year":"2021"},{"key":"B61","doi-asserted-by":"publisher","first-page":"155","DOI":"10.1172\/JCI0214842","article-title":"Membrane Domains and the Immunological Synapse: Keeping T Cells Resting and Ready","volume":"109","author":"Dustin","year":"2002","journal-title":"J. Clin. Invest."},{"key":"B62","doi-asserted-by":"publisher","first-page":"402","DOI":"10.1016\/j.jaci.2014.12.1945","article-title":"The Extended Clinical Phenotype of 64 Patients with Dedicator of Cytokinesis 8 Deficiency","volume":"136","author":"Engelhardt","year":"2015","journal-title":"J. Allergy Clin. Immunol."},{"key":"B63","doi-asserted-by":"publisher","first-page":"1289","DOI":"10.1016\/j.jaci.2009.10.038","article-title":"Large Deletions and point Mutations Involving the Dedicator of Cytokinesis 8 (DOCK8) in the Autosomal-Recessive Form of Hyper-IgE Syndrome","volume":"124","author":"Engelhardt","year":"2009","journal-title":"J. Allergy Clin. Immunol."},{"key":"B64","unstructured":"Rare Diseases2021"},{"key":"B65","doi-asserted-by":"publisher","first-page":"1298","DOI":"10.1038\/s41587-020-0551-y","article-title":"Efficient Gene Editing of Human Long-Term Hematopoietic Stem Cells Validated by Clonal Tracking","volume":"38","author":"Ferrari","year":"2020","journal-title":"Nat. Biotechnol."},{"key":"B66","doi-asserted-by":"publisher","first-page":"917","DOI":"10.1182\/blood-2017-09-807487","article-title":"Successful Outcome Following Allogeneic Hematopoietic Stem Cell Transplantation in Adults with Primary Immunodeficiency","volume":"131","author":"Fox","year":"2018","journal-title":"Blood"},{"key":"B67","doi-asserted-by":"publisher","first-page":"252","DOI":"10.1056\/NEJMoa2031054","article-title":"CRISPR-Cas9 Gene Editing for Sickle Cell Disease and \u03b2-Thalassemia","volume":"384","author":"Frangoul","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"B68","doi-asserted-by":"publisher","first-page":"969","DOI":"10.1093\/nar\/gkaa1251","article-title":"Dynamics and Competition of CRISPR-Cas9 Ribonucleoproteins and AAV Donor-Mediated NHEJ, MMEJ and HDR Editing","volume":"49","author":"Fu","year":"2021","journal-title":"Nucleic Acids Res."},{"key":"B69","doi-asserted-by":"publisher","first-page":"97ra79","DOI":"10.1126\/scitranslmed.3002715","article-title":"Long-term Persistence of a Polyclonal T Cell Repertoire after Gene Therapy for X-Linked Severe Combined Immunodeficiency","volume":"3","author":"Gaspar","year":"2011","journal-title":"Sci. Transl. Med."},{"key":"B70","doi-asserted-by":"publisher","first-page":"2181","DOI":"10.1016\/S0140-6736(04)17590-9","article-title":"Gene Therapy of X-Linked Severe Combined Immunodeficiency by Use of a Pseudotyped Gammaretroviral Vector","volume":"364","author":"Gaspar","year":"2004","journal-title":"The Lancet"},{"key":"B71","doi-asserted-by":"publisher","first-page":"212","DOI":"10.1038\/d41586-021-00314-6","article-title":"A Wealth of Discovery Built on the Human Genome Project - by the Numbers","volume":"590","author":"Gates","year":"2021","journal-title":"Nature"},{"key":"B72","doi-asserted-by":"publisher","first-page":"48","DOI":"10.5500\/wjt.v9.i3.48","article-title":"Considerations for Hematopoietic Stem Cell Transplantation in Primary Immunodeficiency Disorders","volume":"9","author":"Gavrilova","year":"2019","journal-title":"Wjt"},{"key":"B73","doi-asserted-by":"publisher","first-page":"1311","DOI":"10.1038\/s41591-021-01481-9","article-title":"Gene Therapies Should Be for All","volume":"27","author":"Gene","year":"2021","journal-title":"Nat. Med."},{"key":"B74","doi-asserted-by":"publisher","first-page":"235","DOI":"10.1038\/nature13420","article-title":"Targeted Genome Editing in Human Repopulating Haematopoietic Stem Cells","volume":"510","author":"Genovese","year":"2014","journal-title":"Nature"},{"key":"B75","doi-asserted-by":"publisher","first-page":"3056","DOI":"10.1111\/trf.15020","article-title":"Humoral Immunodeficiencies: Conferred Risk of Infections and Benefits of Immunoglobulin Replacement Therapy","volume":"58","author":"Gernez","year":"2018","journal-title":"Transfusion"},{"key":"B76","doi-asserted-by":"publisher","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."},{"key":"B77","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1186\/s13073-017-0494-1","article-title":"Copy Number Variation Meta-Analysis Reveals a Novel Duplication at 9p24 Associated with Multiple Neurodevelopmental Disorders","volume":"9","author":"Glessner","year":"2017","journal-title":"Genome Med."},{"key":"B78","doi-asserted-by":"publisher","first-page":"191","DOI":"10.1089\/crispr.2020.0080","article-title":"Optimizing Integration and Expression of Transgenic Bruton's Tyrosine Kinase for CRISPR-Cas9-Mediated Gene Editing of X-Linked Agammaglobulinemia","volume":"4","author":"Gray","year":"2021","journal-title":"CRISPR J."},{"key":"B79","doi-asserted-by":"publisher","first-page":"9933","DOI":"10.1128\/jvi.79.15.9933-9944.2005","article-title":"Packaging Capacity of Adeno-Associated Virus Serotypes: Impact of Larger Genomes on Infectivity and Postentry Steps","volume":"79","author":"Grieger","year":"2005","journal-title":"J. Virol."},{"key":"B80","doi-asserted-by":"publisher","first-page":"692","DOI":"10.1056\/NEJM199903043400904","article-title":"Hyper-IgE Syndrome with Recurrent Infections - an Autosomal Dominant Multisystem Disorder","volume":"340","author":"Grimbacher","year":"1999","journal-title":"N. Engl. J. Med."},{"key":"B81","doi-asserted-by":"publisher","first-page":"4","DOI":"10.1016\/j.exphem.2017.07.006","article-title":"Technical Considerations for the Use of CRISPR\/Cas9 in Hematology Research","volume":"54","author":"Gundry","year":"2017","journal-title":"Exp. Hematol."},{"key":"B82","doi-asserted-by":"publisher","first-page":"1550","DOI":"10.1001\/jama.2015.3253","article-title":"Outcomes Following Gene Therapy in Patients with Severe Wiskott-Aldrich Syndrome","volume":"313","author":"Hacein-Bey Abina","year":"2015","journal-title":"JAMA"},{"key":"B83","doi-asserted-by":"publisher","first-page":"355","DOI":"10.1056\/NEJMoa1000164","article-title":"Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency","volume":"363","author":"Hacein-Bey-Abina","year":"2010","journal-title":"N. Engl. J. Med."},{"key":"B84","doi-asserted-by":"publisher","first-page":"1185","DOI":"10.1056\/NEJMoa012616","article-title":"Sustained Correction of X-Linked Severe Combined Immunodeficiency by Ex Vivo Gene Therapy","volume":"346","author":"Hacein-Bey-Abina","year":"2002","journal-title":"N. Engl. J. Med."},{"key":"B85","doi-asserted-by":"publisher","first-page":"4451","DOI":"10.1182\/blood-2012-01-407098","article-title":"DOCK8 Is a Cdc42 Activator Critical for Interstitial Dendritic Cell Migration during Immune Responses","volume":"119","author":"Harada","year":"2012","journal-title":"Blood"},{"key":"B86","doi-asserted-by":"publisher","first-page":"515","DOI":"10.1111\/pai.13236","article-title":"Clinical, Immunological Features and Follow up of 20 Patients with Dedicator of Cytokinesis 8 (DOCK8) Deficiency","volume":"31","author":"Haskologlu","year":"2020","journal-title":"Pediatr. Allergy Immunol."},{"key":"B87","doi-asserted-by":"publisher","first-page":"985","DOI":"10.1038\/nbt.3290","article-title":"Chemically Modified Guide RNAs Enhance CRISPR-Cas Genome Editing in Human Primary Cells","volume":"33","author":"Hendel","year":"2015","journal-title":"Nat. Biotechnol."},{"key":"B88","doi-asserted-by":"publisher","first-page":"2513","DOI":"10.1182\/blood-2015-11-683235","article-title":"Targeted Gene Editing Restores Regulated CD40L Function in X-Linked Hyper-IgM Syndrome","volume":"127","author":"Hubbard","year":"2016","journal-title":"Blood"},{"key":"B89","doi-asserted-by":"publisher","first-page":"612","DOI":"10.1038\/ni.2305","article-title":"DOCK8 Functions as an Adaptor that Links TLR-MyD88 Signaling to B Cell Activation","volume":"13","author":"Jabara","year":"2012","journal-title":"Nat. Immunol."},{"key":"B90","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1172\/jci.insight.94298","article-title":"DOCK8 Enforces Immunological Tolerance by Promoting IL-2 Signaling and Immune Synapse Formation in Tregs","volume":"2","author":"Janssen","year":"2017","journal-title":"JCI Insight"},{"key":"B91","doi-asserted-by":"publisher","first-page":"1365","DOI":"10.1016\/j.jaci.2014.07.042","article-title":"Dedicator of Cytokinesis 8-deficient Patients Have a Breakdown in Peripheral B-Cell Tolerance and Defective Regulatory T Cells","volume":"134","author":"Janssen","year":"2014","journal-title":"J. Allergy Clin. Immunol."},{"key":"B92","doi-asserted-by":"publisher","first-page":"5","DOI":"10.1172\/jci.insight.134508","article-title":"DOCK8 Is Essential for LFA-1-dependent Positioning of T Follicular Helper Cells in Germinal Centers","volume":"5","author":"Janssen","year":"2020","journal-title":"JCI Insight"},{"key":"B93","doi-asserted-by":"publisher","first-page":"3837","DOI":"10.1172\/JCI85774","article-title":"A DOCK8-WIP-WASp Complex Links T Cell Receptors to the Actin Cytoskeleton","volume":"126","author":"Janssen","year":"2016","journal-title":"J. Clin. Invest."},{"key":"B94","doi-asserted-by":"publisher","first-page":"73","DOI":"10.1016\/j.jaci.2021.02.025","article-title":"Macabre TH2 Skewing in DOCK8 Deficiency","volume":"148","author":"Janssen","year":"2021","journal-title":"J. Allergy Clin. Immunol."},{"key":"B95","doi-asserted-by":"publisher","first-page":"2866","DOI":"10.1038\/s41467-019-10735-7","article-title":"CRISPR-Cas9 Fusion to Dominant-Negative 53BP1 Enhances HDR and Inhibits NHEJ Specifically at Cas9 Target Sites","volume":"10","author":"Jayavaradhan","year":"2019","journal-title":"Nat. Commun."},{"key":"B96","doi-asserted-by":"publisher","first-page":"821","DOI":"10.1111\/bjh.15851","article-title":"Therapeutic Gene Editing in Haematological Disorders withCRISPR\/Cas9","volume":"185","author":"Jensen","year":"2019","journal-title":"Br. J. Haematol."},{"key":"B97","doi-asserted-by":"publisher","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"},{"key":"B98","doi-asserted-by":"publisher","first-page":"1667","DOI":"10.1016\/j.jaci.2014.03.025","article-title":"Somatic Reversion in Dedicator of Cytokinesis 8 Immunodeficiency Modulates Disease Phenotype","volume":"133","author":"Jing","year":"2014","journal-title":"J. Allergy Clin. Immunol."},{"key":"B99","doi-asserted-by":"publisher","first-page":"2125","DOI":"10.2147\/RMHP.S260641","article-title":"Ethical Questions Linked to Rare Diseases and Orphan Drugs - A Systematic Review","volume":"13","author":"Kacetl","year":"2020","journal-title":"Rmhp"},{"key":"B100","doi-asserted-by":"publisher","first-page":"783","DOI":"10.1182\/blood-2009-05-222760","article-title":"Retrovirus Gene Therapy for X-Linked Chronic Granulomatous Disease Can Achieve Stable Long-Term Correction of Oxidase Activity in Peripheral Blood Neutrophils","volume":"115","author":"Kang","year":"2010","journal-title":"Blood"},{"key":"B101","doi-asserted-by":"publisher","first-page":"1384","DOI":"10.1016\/j.jaci.2016.04.023.DOCK8","article-title":"Dedicator of Cytokinesis 8 Regulates Signal Transducer and Activator of Transcription 3 Activation and Promotes TH17 Cell Differentiation","volume":"138","author":"Keles","year":"2016","journal-title":"J. Allergy Clin. Immunol."},{"key":"B102","doi-asserted-by":"publisher","first-page":"1753","DOI":"10.1016\/j.jaci.2014.03.032","article-title":"Plasmacytoid Dendritic Cell Depletion in DOCK8 Deficiency: Rescue of Severe Herpetic Infections with IFN-\u03b1 2b Therapy","volume":"133","author":"Keles","year":"2014","journal-title":"J. Allergy Clin. Immunol."},{"key":"B103","doi-asserted-by":"publisher","first-page":"1894","DOI":"10.1101\/gr.236620.118","article-title":"DIG-seq: A Genome-wide CRISPR Off-Target Profiling Method Using Chromatin DNA","volume":"28","author":"Kim","year":"2018","journal-title":"Genome Res."},{"key":"B104","doi-asserted-by":"publisher","first-page":"1012","DOI":"10.1101\/gr.171322.113","article-title":"Highly Efficient RNA-Guided Genome Editing in Human Cells via Delivery of Purified Cas9 Ribonucleoproteins","volume":"24","author":"Kim","year":"2014","journal-title":"Genome Res."},{"key":"B105","doi-asserted-by":"publisher","first-page":"1156","DOI":"10.1073\/pnas.93.3.1156","article-title":"Hybrid Restriction Enzymes: Zinc finger Fusions to Fok I Cleavage Domain","volume":"93","author":"Kim","year":"1996","journal-title":"Proc. Natl. Acad. Sci."},{"key":"B106","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1126\/sciimmunol.aam9169","article-title":"Migratory CD11b + Conventional Dendritic Cells Induce T Follicular Helper Cell-dependent Antibody Responses","volume":"2","author":"Krishnaswamy","year":"2017","journal-title":"Sci. Immunol."},{"key":"B107","doi-asserted-by":"publisher","first-page":"3056","DOI":"10.1073\/pnas.1501554112","article-title":"Coincidental Loss of DOCK8 Function in NLRP10-Deficient and C3H\/HeJ Mice Results in Defective Dendritic Cell Migration","volume":"112","author":"Krishnaswamy","year":"2015","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"B108","doi-asserted-by":"publisher","first-page":"546","DOI":"10.1016\/j.bbamem.2013.05.004","article-title":"T Cell Antigen Receptor Activation and Actin Cytoskeleton Remodeling","volume":"1838","author":"Kumari","year":"2014","journal-title":"Biochim. Biophys. Acta (Bba) - Biomembranes"},{"key":"B109","doi-asserted-by":"publisher","first-page":"5","DOI":"10.1093\/intimm\/dxz067","article-title":"DOCK Family Proteins: Key Players in Immune Surveillance Mechanisms","volume":"32","author":"Kunimura","year":"2020","journal-title":"Int. Immunol."},{"key":"B110","doi-asserted-by":"publisher","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":"Cel Rep."},{"key":"B111","doi-asserted-by":"publisher","first-page":"2104","DOI":"10.1182\/blood-2018-06-853879","article-title":"Anti-human CD117 Antibody-Mediated Bone Marrow Niche Clearance in Nonhuman Primates and Humanized NSG Mice","volume":"133","author":"Kwon","year":"2019","journal-title":"Blood"},{"key":"B112","doi-asserted-by":"publisher","first-page":"3423","DOI":"10.1002\/eji.201141759","article-title":"DOCK8 Is Essential for T-Cell Survival and the Maintenance of CD8+ T-Cell Memory","volume":"41","author":"Lambe","year":"2011","journal-title":"Eur. J. Immunol."},{"key":"B113","doi-asserted-by":"publisher","first-page":"165","DOI":"10.1038\/d41586-019-00726-5","article-title":"Adopt a Moratorium on Heritable Genome Editing","volume":"567","author":"Lander","year":"2019","journal-title":"Nature"},{"key":"B114","doi-asserted-by":"publisher","first-page":"13","DOI":"10.1126\/scitranslmed.abf2444","article-title":"Development of \u03b2-globin Gene Correction in Human Hematopoietic Stem Cells as a Potential Durable Treatment for Sickle Cell Disease","volume":"13","author":"Lattanzi","year":"2021","journal-title":"Sci. Transl. Med."},{"key":"B115","first-page":"127","article-title":"Electroporation-Based CRISPR\/Cas9 Gene Editing Using Cas9 Protein and Chemically Modified sgRNAs: Methods and Protocols","volume-title":"Methods in Molecular Biology (Clifton, N.J.)","author":"Laustsen","year":"2019"},{"key":"B116","doi-asserted-by":"publisher","first-page":"860","DOI":"10.1038\/nrm2522","article-title":"Beyond Polymer Polarity: How the Cytoskeleton Builds a Polarized Cell","volume":"9","author":"Li","year":"2008","journal-title":"Nat. Rev. Mol. Cel Biol."},{"key":"B117","first-page":"e04766","article-title":"Enhanced Homology-Directed Human Genome Engineering by Controlled Timing of CRISPR\/Cas9 Delivery","volume-title":"Elife","author":"Lin","year":"2014"},{"key":"B118","doi-asserted-by":"publisher","first-page":"1234","DOI":"10.1080\/10717544.2018.1474964","article-title":"Delivering Crispr: A Review of the Challenges and Approaches","volume":"25","author":"Lino","year":"2018","journal-title":"Drug Deliv."},{"key":"B119","doi-asserted-by":"publisher","first-page":"432716","DOI":"10.1101\/432716","article-title":"High-level Correction of the Sickle Mutation Amplified In Vivo during Erythroid Differentiation","author":"Magis","year":"2019","journal-title":"bioRxiv"},{"key":"B120","doi-asserted-by":"publisher","first-page":"833","DOI":"10.1038\/nbt.2675","article-title":"CAS9 Transcriptional Activators for Target Specificity Screening and Paired Nickases for Cooperative Genome Engineering","volume":"31","author":"Mali","year":"2013","journal-title":"Nat. Biotechnol."},{"key":"B121","doi-asserted-by":"publisher","first-page":"539","DOI":"10.1101\/gr.145441.112","article-title":"Obligate Ligation-Gated Recombination (ObLiGaRe): Custom-Designed Nuclease-Mediated Targeted Integration through Nonhomologous End Joining","volume":"23","author":"Maresca","year":"2013","journal-title":"Genome Res."},{"key":"B122","doi-asserted-by":"publisher","first-page":"121","DOI":"10.2217\/cer-2018-0100","article-title":"Cost Associated with Hematopoietic Stem Cell Transplantation: A Retrospective Claims Data Analysis in Germany","volume":"8","author":"Mayerhoff","year":"2019","journal-title":"J. Comp. Effectiveness Res."},{"key":"B123","doi-asserted-by":"publisher","first-page":"151","DOI":"10.3233\/DMA-2010-0740","article-title":"DOCK8 Immune Deficiency as a Model for Primary Cytoskeletal Dysfunction","volume":"29","author":"McGhee","year":"2010","journal-title":"Dis. Markers"},{"key":"B124","doi-asserted-by":"publisher","first-page":"36","DOI":"10.1016\/j.clim.2018.07.011","article-title":"Diagnosis of DOCK8 Deficiency Using Flow Cytometry Biomarkers: an Egyptian Center Experience","volume":"195","author":"Meshaal","year":"2018","journal-title":"Clin. Immunol."},{"key":"B125","doi-asserted-by":"publisher","first-page":"957","DOI":"10.1016\/j.jaci.2016.08.003","article-title":"Exome and Genome Sequencing for Inborn Errors of Immunity","volume":"138","author":"Meyts","year":"2016","journal-title":"J. Allergy Clin. Immunol."},{"key":"B126","doi-asserted-by":"publisher","first-page":"778","DOI":"10.1038\/nbt1319","article-title":"An Improved Zinc-finger Nuclease Architecture for Highly Specific Genome Editing","volume":"25","author":"Miller","year":"2007","journal-title":"Nat. Biotechnol."},{"key":"B127","doi-asserted-by":"publisher","first-page":"143","DOI":"10.1038\/nbt.1755","article-title":"A TALE Nuclease Architecture for Efficient Genome Editing","volume":"29","author":"Miller","year":"2011","journal-title":"Nat. Biotechnol."},{"key":"B128","doi-asserted-by":"publisher","first-page":"773","DOI":"10.1038\/nature06764","article-title":"Impaired TH17 Cell Differentiation in Subjects with Autosomal Dominant Hyper-IgE Syndrome","volume":"452","author":"Milner","year":"2008","journal-title":"Nature"},{"key":"B129","doi-asserted-by":"publisher","first-page":"840","DOI":"10.1016\/j.jaci.2012.12.1568","article-title":"Defective Actin Accumulation Impairs Human Natural Killer Cell Function in Patients with Dedicator of Cytokinesis 8 Deficiency","volume":"131","author":"Mizesko","year":"2013","journal-title":"J. Allergy Clin. Immunol."},{"key":"B130","doi-asserted-by":"publisher","first-page":"1919","DOI":"10.1038\/mt.2009.179","article-title":"Insertional Transformation of Hematopoietic Cells by Self-Inactivating Lentiviral and Gammaretroviral Vectors","volume":"17","author":"Modlich","year":"2009","journal-title":"Mol. Ther."},{"key":"B131","doi-asserted-by":"publisher","first-page":"174","DOI":"10.1016\/j.stem.2010.06.014","article-title":"Hematopoietic Stem Cell Quiescence Promotes Error-Prone DNA Repair and Mutagenesis","volume":"7","author":"Mohrin","year":"2010","journal-title":"Cell Stem Cell."},{"key":"B132","doi-asserted-by":"publisher","first-page":"574","DOI":"10.1016\/j.stem.2017.10.010","article-title":"Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned","volume":"21","author":"Morgan","year":"2017","journal-title":"Cell Stem Cell."},{"key":"B133","doi-asserted-by":"publisher","first-page":"9283","DOI":"10.1093\/nar\/gkr597","article-title":"A Novel TALE Nuclease Scaffold Enables High Genome Editing Activity in Combination with Low Toxicity","volume":"39","author":"Mussolino","year":"2011","journal-title":"Nucleic Acids Res."},{"key":"B134","doi-asserted-by":"publisher","first-page":"3395","DOI":"10.1038\/s41467-019-11105-z","article-title":"Stimulation of CRISPR-Mediated Homology-Directed Repair by an Engineered RAD18 Variant","volume":"10","author":"Nambiar","year":"2019","journal-title":"Nat. Commun."},{"key":"B135","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1016\/j.preteyeres.2014.06.005","article-title":"Dock GEFs and Their Therapeutic Potential: Neuroprotection and Axon Regeneration","volume":"43","author":"Namekata","year":"2014","journal-title":"Prog. Retin. Eye Res."},{"key":"B136","doi-asserted-by":"publisher","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":"B137","doi-asserted-by":"publisher","first-page":"401","DOI":"10.1038\/nm1393","article-title":"Correction of X-Linked Chronic Granulomatous Disease by Gene Therapy, Augmented by Insertional Activation of MDS1-EVI1, PRDM16 or SETBP1","volume":"12","author":"Ott","year":"2006","journal-title":"Nat. Med."},{"key":"B138","doi-asserted-by":"publisher","first-page":"1456","DOI":"10.1016\/j.jaci.2014.02.008","article-title":"Beneficial IFN-\u03b1 Treatment of Tumorous Herpes Simplex Blepharoconjunctivitis in Dedicator of Cytokinesis 8 Deficiency","volume":"133","author":"Papan","year":"2014","journal-title":"J. Allergy Clin. Immunol."},{"key":"B139","doi-asserted-by":"publisher","first-page":"1634","DOI":"10.1038\/s41467-019-09614-y","article-title":"Gene Correction for SCID-X1 in Long-Term Hematopoietic Stem Cells","volume":"10","author":"Pavel-Dinu","year":"2019","journal-title":"Nat. Commun."},{"key":"B140","doi-asserted-by":"publisher","first-page":"131","DOI":"10.1172\/JCI142434","article-title":"Somatic Reversion of Pathogenic DOCK8 Variants Alters Lymphocyte Differentiation and Function to Effectively Cure DOCK8 Deficiency","volume":"131","author":"Pillay","year":"2021","journal-title":"J. Clin. Invest."},{"key":"B141","doi-asserted-by":"publisher","first-page":"947","DOI":"10.1056\/nejmra1800729","article-title":"A New Class of Medicines through DNA Editing","volume":"380","author":"Porteus","year":"2019","journal-title":"N. Engl. J. Med."},{"key":"B142","doi-asserted-by":"publisher","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."},{"key":"B143","doi-asserted-by":"publisher","first-page":"1283","DOI":"10.1038\/ni.1820.DOCK8","article-title":"Europe PMC Funders Group DOCK8 Mutations Cripple B Cell Immune Synapse, Germinal Centers and Long-Lived Antibody Production","volume":"10","author":"Randall","year":"2012","journal-title":"Nat. Immunol."},{"key":"B144","doi-asserted-by":"publisher","first-page":"2305","DOI":"10.1084\/jem.20110345","article-title":"DOCK8 Deficiency Impairs CD8 T Cell Survival and Function in Humans and Mice","volume":"208","author":"Randall","year":"2011","journal-title":"J. Exp. Med."},{"key":"B145","doi-asserted-by":"publisher","first-page":"1283","DOI":"10.1038\/ni.1820","article-title":"Dock8 Mutations Cripple B Cell Immunological Synapses, Germinal Centers and Long-Lived Antibody Production","volume":"10","author":"Randall","year":"2009","journal-title":"Nat. Immunol."},{"key":"B146","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1002\/cti2.1236","article-title":"DOCK8 Deficiency Diminishes Thymic T\u2010regulatory Cell Development but Not Thymic Deletion","volume":"10","author":"Randall","year":"2021","journal-title":"Clin. Transl Immunol."},{"key":"B147","doi-asserted-by":"publisher","first-page":"93","DOI":"10.1186\/s13073-015-0215-6","article-title":"Pharmacological Inhibition of DNA-PK Stimulates Cas9-Mediated Genome Editing","volume":"7","author":"Robert","year":"2015","journal-title":"Genome Med."},{"key":"B148","doi-asserted-by":"publisher","first-page":"607","DOI":"10.1016\/j.stem.2019.08.016","article-title":"NHEJ-mediated Repair of CRISPR-Cas9-Induced DNA Breaks Efficiently Corrects Mutations in HSPCs from Patients with Fanconi Anemia","volume":"25","author":"Rom\u00e1n-Rodr\u00edguez","year":"2019","journal-title":"Cell Stem Cell."},{"key":"B149","doi-asserted-by":"publisher","first-page":"1389","DOI":"10.1016\/j.ymthe.2019.05.014","article-title":"Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates","volume":"27","author":"Romero","year":"2019","journal-title":"Mol. Ther."},{"key":"B150","doi-asserted-by":"publisher","first-page":"405","DOI":"10.1038\/s41586-018-0326-5","article-title":"Reprogramming Human T Cell Function and Specificity with Non-viral Genome Targeting","volume":"559","author":"Roth","year":"2018","journal-title":"Nature"},{"key":"B151","doi-asserted-by":"publisher","first-page":"118","DOI":"10.1038\/nprot.2015.140","article-title":"MMEJ-assisted Gene Knock-In Using TALENs and CRISPR-Cas9 with the PITCh Systems","volume":"11","author":"Sakuma","year":"2016","journal-title":"Nat. Protoc."},{"key":"B152","doi-asserted-by":"publisher","first-page":"e202000873","DOI":"10.26508\/lsa.202000873","article-title":"A Conserved PI(4,5)P2-binding Domain Is Critical for Immune Regulatory Function of DOCK8","volume":"4","author":"Sakurai","year":"2021","journal-title":"Life Sci. Alliance"},{"key":"B153","doi-asserted-by":"publisher","first-page":"205","DOI":"10.12688\/f1000research.13020.1","article-title":"Update on Regulation and Effector Functions of Th17 Cells","volume":"7","author":"Sandquist","year":"2018","journal-title":"F1000Res"},{"key":"B154","doi-asserted-by":"publisher","first-page":"551","DOI":"10.1016\/j.stem.2019.02.019","article-title":"Precise Gene Editing Preserves Hematopoietic Stem Cell Function Following Transient P53-Mediated DNA Damage Response","volume":"24","author":"Schiroli","year":"2019","journal-title":"Cell Stem Cell."},{"key":"B155","doi-asserted-by":"publisher","first-page":"396","DOI":"10.1038\/s41434-021-00263-9","article-title":"Prime Editing - an Update on the Field","volume":"28","author":"Scholefield","year":"2021","journal-title":"Gene Ther."},{"key":"B156","doi-asserted-by":"publisher","first-page":"582","DOI":"10.1007\/s10875-017-0421-7","article-title":"Long-Term Outcome of Adenosine Deaminase-Deficient Patients-A Single-Center Experience","volume":"37","author":"Scott","year":"2017","journal-title":"J. Clin. Immunol."},{"key":"B157","doi-asserted-by":"publisher","first-page":"899","DOI":"10.1016\/j.immuni.2018.10.010","article-title":"Hippo Kinases Mst1 and Mst2 Sense and Amplify IL-2R-STAT5 Signaling in Regulatory T Cells to Establish Stable Regulatory Activity","volume":"49","author":"Shi","year":"2018","journal-title":"Immunity"},{"key":"B158","doi-asserted-by":"publisher","first-page":"108093","DOI":"10.1016\/j.celrep.2020.108093","article-title":"Controlled Cycling and Quiescence Enables Efficient HDR in Engraftment-Enriched Adult Hematopoietic Stem and Progenitor Cells","volume":"32","author":"Shin","year":"2020","journal-title":"Cel Rep."},{"key":"B159","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1172\/jci.insight.94275","article-title":"DOCK8 Regulates Fitness and Function of Regulatory T Cells through Modulation of IL-2 Signaling","volume":"2","author":"Singh","year":"2017","journal-title":"JCI Insight"},{"key":"B160","doi-asserted-by":"publisher","first-page":"e118","DOI":"10.1093\/nar\/gkv575","article-title":"Cas9-chromatin Binding Information Enables More Accurate CRISPR Off-Target Prediction","volume":"43","author":"Singh","year":"2015","journal-title":"Nucleic Acids Res."},{"key":"B161","doi-asserted-by":"publisher","first-page":"103","DOI":"10.1080\/1744666X.2018.1424627","article-title":"Hematopoietic Cell Transplantation in Primary Immunodeficiency - Conventional and Emerging Indications","volume":"14","author":"Slatter","year":"2018","journal-title":"Expert Rev. Clin. Immunol."},{"key":"B162","doi-asserted-by":"publisher","first-page":"10548","DOI":"10.1038\/ncomms10548","article-title":"RS-1 Enhances CRISPR\/Cas9- and TALEN-Mediated Knock-In Efficiency","volume":"7","author":"Song","year":"2016","journal-title":"Nat. Commun."},{"key":"B163","doi-asserted-by":"publisher","first-page":"367","DOI":"10.1126\/science.aba7365","article-title":"CRISPR-engineered T Cells in Patients with Refractory Cancer","volume":"367","author":"Stadtmauer","year":"2020","journal-title":"Science"},{"key":"B164","doi-asserted-by":"publisher","first-page":"9","DOI":"10.1111\/imr.12723","article-title":"Insights into Immunity from Clinical and Basic Science Studies of DOCK8 Immunodeficiency Syndrome","volume":"287","author":"Su","year":"2019","journal-title":"Immunol. Rev."},{"key":"B165","doi-asserted-by":"publisher","first-page":"26","DOI":"10.1111\/j.1749-6632.2011.06295.x","article-title":"DOCK8 Deficiency","volume":"1246","author":"Su","year":"2011","journal-title":"Ann. N. Y Acad. Sci."},{"key":"B166","doi-asserted-by":"publisher","first-page":"551","DOI":"10.1007\/s10875-020-00785-8","article-title":"The Growing Spectrum of Human Diseases Caused by InheritedCDC42 Mutations","volume":"40","author":"Su","year":"2020","journal-title":"J. Clin. Immunol."},{"key":"B167","doi-asserted-by":"publisher","first-page":"157","DOI":"10.1038\/s10038-017-0352-4","article-title":"In Vivo genome Editing via the HITI Method as a Tool for Gene Therapy","volume":"63","author":"Suzuki","year":"2018","journal-title":"J. Hum. Genet."},{"key":"B168","doi-asserted-by":"publisher","first-page":"373","DOI":"10.1038\/s41434-021-00251-z","article-title":"Correction of X-CGD Patient HSPCs by Targeted CYBB cDNA Insertion Using CRISPR\/Cas9 with 53BP1 Inhibition for Enhanced Homology-Directed Repair","volume":"28","author":"Sweeney","year":"2021","journal-title":"Gene Ther."},{"key":"B169","doi-asserted-by":"publisher","first-page":"786","DOI":"10.1038\/nbt1317","article-title":"Structure-based Redesign of the Dimerization Interface Reduces the Toxicity of Zinc-finger Nucleases","volume":"25","author":"Szczepek","year":"2007","journal-title":"Nat. Biotechnol."},{"key":"B170","doi-asserted-by":"publisher","first-page":"420","DOI":"10.1002\/sctm.19-0375","article-title":"Unleashing the Cure: Overcoming Persistent Obstacles in the Translation and Expanded Use of Hematopoietic Stem Cell-Based Therapies","volume":"9","author":"Talib","year":"2020","journal-title":"Stem Cell Transl Med"},{"key":"B171","doi-asserted-by":"publisher","first-page":"e12759","DOI":"10.1111\/sji.12759","article-title":"Skewed B Cell Receptor Repertoire and Reduced Antibody Avidity in Patients with DOCK8 Deficiency","volume":"89","author":"Tang","year":"2019","journal-title":"Scand. J. Immunol."},{"key":"B172","doi-asserted-by":"publisher","first-page":"24","DOI":"10.1007\/s10875-019-00737-x","article-title":"Human Inborn Errors of Immunity: 2019 Update on the Classification from the International Union of Immunological Societies Expert Committee","volume":"40","author":"Tangye","year":"2020","journal-title":"J. Clin. Immunol."},{"key":"B173","doi-asserted-by":"publisher","first-page":"933","DOI":"10.1016\/j.jaci.2016.07.016","article-title":"Dedicator of Cytokinesis 8-deficient CD4 + T Cells Are Biased to a T H 2 Effector Fate at the Expense of T H 1 and T H 17 Cells","volume":"139","author":"Tangye","year":"2017","journal-title":"J. Allergy Clin. Immunol."},{"key":"B174","doi-asserted-by":"publisher","first-page":"901","DOI":"10.1056\/NEJMoa1300662","article-title":"Gene Editing ofCCR5in Autologous CD4 T Cells of Persons Infected with HIV","volume":"370","author":"Tebas","year":"2014","journal-title":"N. Engl. J. Med."},{"key":"B175","doi-asserted-by":"publisher","first-page":"187","DOI":"10.1038\/nbt.3117","article-title":"GUIDE-seq Enables Genome-wide Profiling of Off-Target Cleavage by CRISPR-Cas Nucleases","volume":"33","author":"Tsai","year":"2015","journal-title":"Nat. Biotechnol."},{"key":"B176","doi-asserted-by":"publisher","first-page":"864","DOI":"10.1007\/s10875-021-01051-1","article-title":"STAT3 Hyper-IgE Syndrome-An Update and Unanswered Questions","volume":"41","author":"Tsilifis","year":"2021","journal-title":"J. Clin. Immunol."},{"key":"B177","doi-asserted-by":"publisher","first-page":"1136","DOI":"10.1016\/j.stem.2021.02.002","article-title":"Quantitative Evaluation of Chromosomal Rearrangements in Gene-Edited Human Stem Cells by CAST-Seq","volume":"28","author":"Turchiano","year":"2021","journal-title":"Cell Stem Cell."},{"key":"B178","doi-asserted-by":"publisher","first-page":"768","DOI":"10.1038\/s41588-021-00881-4","article-title":"CRISPR-Cas9 Can Cause Chromothripsis","volume":"53","author":"Urnov","year":"2021","journal-title":"Nat. Genet."},{"key":"B179","doi-asserted-by":"publisher","first-page":"530","DOI":"10.1016\/j.omtn.2018.06.010","article-title":"Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification","volume":"12","author":"Vaidyanathan","year":"2018","journal-title":"Mol. Ther. - Nucleic Acids"},{"key":"B180","doi-asserted-by":"publisher","DOI":"10.1101\/2021.02.26.432961","article-title":"Targeted Replacement of Full-Length CFTR in Human Airway Stem Cells by CRISPR\/Cas9 for Pan-Mutation Correction in the Endogenous Locus","author":"Vaidyanathan","year":"2021","journal-title":"bioRxiv"},{"key":"B181","doi-asserted-by":"publisher","first-page":"1216","DOI":"10.1038\/s41591-018-0137-0","article-title":"A High-Fidelity Cas9 Mutant Delivered as a Ribonucleoprotein Complex Enables Efficient Gene Editing in Human Hematopoietic Stem and Progenitor Cells","volume":"24","author":"Vakulskas","year":"2018","journal-title":"Nat. Med."},{"key":"B182","doi-asserted-by":"publisher","first-page":"1","DOI":"10.15252\/emmm.202013545","article-title":"Modeling, Optimization, and Comparable Efficacy of T Cell and Hematopoietic Stem Cell Gene Editing for Treating hyper\u2010IgM Syndrome","volume":"13","author":"Vavassori","year":"2021","journal-title":"EMBO Mol. Med."},{"key":"B183","doi-asserted-by":"publisher","first-page":"1256","DOI":"10.1038\/nbt.3408","article-title":"Homology-driven Genome Editing in Hematopoietic Stem and Progenitor Cells Using ZFN mRNA and AAV6 Donors","volume":"33","author":"Wang","year":"2015","journal-title":"Nat. Biotechnol."},{"key":"B184","doi-asserted-by":"publisher","first-page":"286","DOI":"10.1126\/science.aav9023","article-title":"Unbiased Detection of CRISPR Off-Targets In Vivo Using DISCOVER-Seq","volume":"364","author":"Wienert","year":"2019","journal-title":"Science"},{"key":"B185","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1038\/s41467-021-20909-x","article-title":"Cas9-AAV6 Gene Correction of Beta-Globin in Autologous HSCs Improves Sickle Cell Disease Erythropoiesis in Mice","volume":"12","author":"Wilkinson","year":"2021","journal-title":"Nat. Commun."},{"key":"B186","doi-asserted-by":"publisher","first-page":"1118","DOI":"10.1016\/j.cell.2008.10.048","article-title":"Hematopoietic Stem Cells Reversibly Switch from Dormancy to Self-Renewal during Homeostasis and Repair","volume":"135","author":"Wilson","year":"2008","journal-title":"Cell."},{"key":"B187","doi-asserted-by":"publisher","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."},{"key":"B188","doi-asserted-by":"publisher","first-page":"209","DOI":"10.1084\/jem.20160068","article-title":"LRCH1 Interferes with DOCK8-Cdc42-Induced T Cell Migration and Ameliorates Experimental Autoimmune Encephalomyelitis","volume":"214","author":"Xu","year":"2017","journal-title":"J. Exp. Med."},{"key":"B189","doi-asserted-by":"publisher","first-page":"8","DOI":"10.1038\/ncomms13946","article-title":"The Transcription Factor EPAS1 Links DOCK8 Deficiency to Atopic Skin Inflammation via IL-31 Induction","volume":"8","author":"Yamamura","year":"2017","journal-title":"Nat. Commun."},{"key":"B190","doi-asserted-by":"publisher","first-page":"801","DOI":"10.1038\/cr.2017.76","article-title":"Homology-mediated End Joining-Based Targeted Integration Using CRISPR\/Cas9","volume":"27","author":"Yao","year":"2017","journal-title":"Cell Res."},{"key":"B191","doi-asserted-by":"publisher","first-page":"131","DOI":"10.3233\/DMA-2010-0737","article-title":"Genetic, Clinical, and Laboratory Markers for DOCK8 Immunodeficiency Syndrome","volume":"29","author":"Zhang","year":"2010","journal-title":"Dis. Markers"},{"key":"B192","doi-asserted-by":"publisher","first-page":"2046","DOI":"10.1056\/nejmoa0905506","article-title":"Combined Immunodeficiency Associated withDOCK8Mutations","volume":"361","author":"Zhang","year":"2009","journal-title":"N. Engl. J. Med."},{"key":"B193","doi-asserted-by":"publisher","first-page":"2549","DOI":"10.1084\/jem.20141307","article-title":"DOCK8 Regulates Lymphocyte Shape Integrity for Skin Antiviral Immunity","volume":"211","author":"Zhang","year":"2014","journal-title":"J. Exp. Med."},{"key":"B194","doi-asserted-by":"publisher","first-page":"8","DOI":"10.1111\/imr.12396","article-title":"Regulation of Germinal center B-Cell Differentiation","volume":"270","author":"Zhang","year":"2016","journal-title":"Immunol. Rev."}],"container-title":["Frontiers in Genome Editing"],"original-title":[],"link":[{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fgeed.2022.793010\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,3,17]],"date-time":"2022-03-17T09:02:19Z","timestamp":1647507739000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fgeed.2022.793010\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,3,17]]},"references-count":194,"alternative-id":["10.3389\/fgeed.2022.793010"],"URL":"https:\/\/doi.org\/10.3389\/fgeed.2022.793010","relation":{},"ISSN":["2673-3439"],"issn-type":[{"value":"2673-3439","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,3,17]]},"article-number":"793010"}}