Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease

Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease

Gene editing with the CRISPR-Cas9 system could revolutionize hematopoietic stem cell (HSC)-targeted gene therapy for hereditary diseases, including sickle cell disease (SCD). Conventional delivery of editing tools by electroporation limits HSC fitness due to its toxicity; therefore, efficient and no...

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Bibliographic Details
Main Authors: Naoya Uchida, Claire M. Drysdale, Tina Nassehi, Jackson Gamer, Morgan Yapundich, Julia DiNicola, Yoshitaka Shibata, Malikiya Hinds, Bjorg Gudmundsdottir, Juan J. Haro-Mora, Selami Demirci, John F. Tisdale
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
lentiviral vector
protein delivery
genome editing
CRISPR-Cas9
sickle cell disease
gene correction
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121000371

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http://www.sciencedirect.com/science/article/pii/S2329050121000371

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