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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Bibliographic Details
Main Authors: Rajeev Rai, Asma Naseem, Winston Vetharoy, Zohar Steinberg, Adrian J. Thrasher, Giorgia Santilli, Alessia Cavazza
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123000244