Challenges in Gene Therapy for Somatic Reverted Mosaicism in X-Linked Combined Immunodeficiency by CRISPR/Cas9 and Prime Editing

X-linked severe combined immunodeficiency (X-SCID) is a primary immunodeficiency that is caused by mutations in the interleukin-2 receptor gamma (IL2RG) gene. Some patients present atypical X-SCID with mild clinical symptoms due to somatic revertant mosaicism. CRISPR/Cas9 and prime editing are two a...

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Detalles Bibliográficos
Autores principales: Hou, Yujuan, Ureña-Bailén, Guillermo, Mohammadian Gol, Tahereh, Gratz, Paul Gerhard, Gratz, Hans Peter, Roig-Merino, Alicia, Antony, Justin S., Lamsfus-Calle, Andrés, Daniel-Moreno, Alberto, Handgretinger, Rupert, Mezger, Markus
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9777626/
https://www.ncbi.nlm.nih.gov/pubmed/36553615
http://dx.doi.org/10.3390/genes13122348

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