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Additional CRISPR\/Cas9-driven non-homologous end-joining precision deletion steps allowed correction of 41% of the factor V gene mutated cells, giving rise to a newly developed functional protein. Taking into account the plasma concentrations corresponding to the different levels of severity of factor V deficiency, it may be argued that the correction achieved in this study could, in ideal conditions, be sufficient to turn a severe phenotype into a mild or asymptomatic one.<\/jats:p>","DOI":"10.3390\/ijms23105802","type":"journal-article","created":{"date-parts":[[2022,5,22]],"date-time":"2022-05-22T07:13:57Z","timestamp":1653203637000},"page":"5802","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Development and Characterization of a Factor V-Deficient CRISPR Cell Model for the Correction of Mutations"],"prefix":"10.3390","volume":"23","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0363-3148","authenticated-orcid":false,"given":"Luis Javier","family":"Serrano","sequence":"first","affiliation":[{"name":"Department of Genetics, Physiology and Microbiology, School of Biology, Complutense University, 28040 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mariano","family":"Garcia-Arranz","sequence":"additional","affiliation":[{"name":"Fundaci\u00f3n Jim\u00e9nez D\u00edaz University Hospital Health Research Institute, Fundaci\u00f3n Jim\u00e9nez D\u00edaz University Hospital, 28040 Madrid, Spain"},{"name":"Department of Surgery, Autonomous University of Madrid, 28049 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9491-5572","authenticated-orcid":false,"given":"Juan A.","family":"De Pablo-Moreno","sequence":"additional","affiliation":[{"name":"Department of Genetics, Physiology and Microbiology, School of Biology, Complutense University, 28040 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jos\u00e9 Carlos","family":"Segovia","sequence":"additional","affiliation":[{"name":"Differentiation and Cytometry Unit, Innovative Hematopoietic Therapies Division, Center for Energy, Environmental and Technological Research (CIEMAT), Biomedical Research Networking Center for Rare Diseases (CIBERER), 28040 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Roc\u00edo","family":"Olivera-Salazar","sequence":"additional","affiliation":[{"name":"Fundaci\u00f3n Jim\u00e9nez D\u00edaz University Hospital Health Research Institute, Fundaci\u00f3n Jim\u00e9nez D\u00edaz University Hospital, 28040 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9369-2338","authenticated-orcid":false,"given":"Dami\u00e1n","family":"Garcia-Olmo","sequence":"additional","affiliation":[{"name":"Fundaci\u00f3n Jim\u00e9nez D\u00edaz University Hospital Health Research Institute, Fundaci\u00f3n Jim\u00e9nez D\u00edaz University Hospital, 28040 Madrid, Spain"},{"name":"Department of Surgery, Autonomous University of Madrid, 28049 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Antonio","family":"Liras","sequence":"additional","affiliation":[{"name":"Department of Genetics, Physiology and Microbiology, School of Biology, Complutense University, 28040 Madrid, Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2022,5,22]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"530","DOI":"10.1160\/TH07-02-0150","article-title":"Coagulation factor V and thrombophilia: Background and mechanisms","volume":"98","author":"Segers","year":"2007","journal-title":"Thromb. 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