An improved medium formulation for efficient ex vivo gene editing, expansion and engraftment of hematopoietic stem and progenitor cells

Gene editing has emerged as a powerful tool for the therapeutic correction of monogenic diseases. CRISPR-Cas9 applied to hematopoietic stem and progenitor cells (HSPCs) has shown great promise in proof-of-principle preclinical studies to treat hematological disorders, and clinical trials using these...

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Detalles Bibliográficos
Autores principales: Rai, Rajeev, Naseem, Asma, Vetharoy, Winston, Steinberg, Zohar, Thrasher, Adrian J., Santilli, Giorgia, Cavazza, Alessia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10025975/
https://www.ncbi.nlm.nih.gov/pubmed/36950452
http://dx.doi.org/10.1016/j.omtm.2023.02.014

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