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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Vydáno v:Mol Ther Methods Clin Dev
Hlavní autoři: Uchida, Naoya, Drysdale, Claire M., Nassehi, Tina, Gamer, Jackson, Yapundich, Morgan, DiNicola, Julia, Shibata, Yoshitaka, Hinds, Malikiya, Gudmundsdottir, Bjorg, Haro-Mora, Juan J., Demirci, Selami, Tisdale, John F.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2021
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC8005818/
https://ncbi.nlm.nih.gov/pubmed/33816645
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.02.022
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