CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells

INTRODUCTION: Ex vivo gene therapy for treatment of Inborn errors of Immunity (IEIs) have demonstrated significant clinical benefit in multiple Phase I/II clinical trials. Current approaches rely on engineered retroviral vectors to randomly integrate copy(s) of gene-of-interest in autologous hematop...

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Detalles Bibliográficos
Autores principales: Brault, Julie, Liu, Taylor, Liu, Siyuan, Lawson, Amanda, Choi, Uimook, Kozhushko, Nikita, Bzhilyanskaya, Vera, Pavel-Dinu, Mara, Meis, Ronald J., Eckhaus, Michael A., Burkett, Sandra S., Bosticardo, Marita, Kleinstiver, Benjamin P., Notarangelo, Luigi D., Lazzarotto, Cicera R., Tsai, Shengdar Q., Wu, Xiaolin, Dahl, Gary A., Porteus, Matthew H., Malech, Harry L., De Ravin, Suk See
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Immunology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9846165/
https://www.ncbi.nlm.nih.gov/pubmed/36685559
http://dx.doi.org/10.3389/fimmu.2022.1067417

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