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Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivo
Hyper-IgM1 is a rare X-linked combined immunodeficiency caused by mutations in the CD40 ligand (CD40LG) gene with a median survival of 25 years, potentially treatable with in situ CD4+ T cell gene editing with Cas9 and a one-size-fits-most corrective donor template. Here, starting from our research-...
Autores principales: | , , , , , , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10482894/ https://www.ncbi.nlm.nih.gov/pubmed/37693944 http://dx.doi.org/10.1016/j.omtm.2023.08.020 |