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Genome Editing With TALEN, CRISPR-Cas9 and CRISPR-Cas12a in Combination With AAV6 Homology Donor Restores T Cell Function for XLP

X-linked lymphoproliferative disease is a rare inherited immune disorder, caused by mutations or deletions in the SH2D1A gene that encodes an intracellular adapter protein SAP (Slam-associated protein). SAP is essential for mediating several key immune processes and the immune system - T cells in pa...

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Detalles Bibliográficos
Autores principales: Houghton, Benjamin C., Panchal, Neelam, Haas, Simone A., Chmielewski, Kay O., Hildenbeutel, Markus, Whittaker, Thomas, Mussolino, Claudio, Cathomen, Toni, Thrasher, Adrian J, Booth, Claire
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9168036/
https://www.ncbi.nlm.nih.gov/pubmed/35677600
http://dx.doi.org/10.3389/fgeed.2022.828489