In Vivo Outcome of Homology-Directed Repair at the HBB Gene in HSC Using Alternative Donor Template Delivery Methods

Gene editing following designer nuclease cleavage in the presence of a DNA donor template can revert mutations in disease-causing genes. For optimal benefit, reversion of the point mutation in HBB leading to sickle cell disease (SCD) would permit precise homology-directed repair (HDR) while concurre...

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Detalles Bibliográficos
Autores principales: Pattabhi, Sowmya, Lotti, Samantha N., Berger, Mason P., Singh, Swati, Lux, Christopher T., Jacoby, Kyle, Lee, Calvin, Negre, Olivier, Scharenberg, Andrew M., Rawlings, David J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6611979/
https://www.ncbi.nlm.nih.gov/pubmed/31279229
http://dx.doi.org/10.1016/j.omtn.2019.05.025

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