Biallelic modification of IL2RG leads to severe combined immunodeficiency in pigs

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dc.contributor.authorClark-Deener, Sherrieen
dc.contributor.authorKang, Jung-Taeken
dc.contributor.authorCho, Bumraeen
dc.contributor.authorRyu, Junghyunen
dc.contributor.authorRay, Caitlinen
dc.contributor.authorLee, Eun-Jinen
dc.contributor.authorAhn, SunMien
dc.contributor.authorLee, JinSeoken
dc.contributor.authorJi, Dal-Youngen
dc.contributor.authorJue, Nathanielen
dc.contributor.authorLee, Kihoen
dc.contributor.authorPark, Kwang-Wooken
dc.contributor.departmentAnimal and Poultry Sciencesen
dc.contributor.departmentLarge Animal Clinical Sciencesen
dc.date.accessioned2017-01-02T18:34:44Zen
dc.date.available2017-01-02T18:34:44Zen
dc.date.issued2016-11-03en
dc.description.abstractBackground: Pigs with SCID can be a useful model in regenerative medicine, xenotransplantation, and cancer cell transplantation studies. Utilizing genome editing technologies such as CRISPR/Cas9 system allows us to generate genetically engineered pigs at a higher efficiency. In this study, we report generation and phenotypic characterization of IL2RG knockout female pigs produced through combination of CRISPR/Cas9 system and SCNT. As expected, pigs lacking IL2RG presented SCID phenotype. Methods: First, specific CRISPR/Cas9 systems targeting IL2RG were introduced into developing pig embryos then the embryos were transferred into surrogates. A total of six fetuses were obtained from the embryo transfer and fetal fibroblast cell lines were established. Then IL2RG knockout female cells carrying biallelic genetic modification were used as donor cells for SCNT, followed by embryo transfer. Results: Three live cloned female piglets carrying biallelic mutations in IL2RG were produced. All cloned piglets completely lacked thymus and they had a significantly reduced level of mature T, B and NK cells in their blood and spleen. Conclusions: Here, we generated IL2RG knockout female pigs showing phenotypic characterization of SCID. This IL2RG knockout female pigs will be a promising model for biomedical and translational research.en
dc.description.versionPublished versionen
dc.format.extent74 - 74 page(s)en
dc.format.mimetypeapplication/pdfen
dc.identifier.doihttps://doi.org/10.1186/s12958-016-0206-5en
dc.identifier.issue1en
dc.identifier.urihttp://hdl.handle.net/10919/73918en
dc.identifier.volume14en
dc.language.isoenen
dc.rightsCreative Commons Attribution 4.0 Internationalen
dc.rights.holderThe Author(s)en
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/en
dc.titleBiallelic modification of IL2RG leads to severe combined immunodeficiency in pigsen
dc.title.serialReproductive Biology and Endocrinologyen
dc.typeArticle - Refereeden
dc.type.dcmitypeTexten
pubs.organisational-group/Virginia Techen
pubs.organisational-group/Virginia Tech/All T&R Facultyen
pubs.organisational-group/Virginia Tech/Veterinary Medicineen
pubs.organisational-group/Virginia Tech/Veterinary Medicine/CVM T&R Facultyen
pubs.organisational-group/Virginia Tech/Veterinary Medicine/Large Animal Clinical Sciencesen

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