CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich’s Ataxia

Friedreich’s ataxia (FRDA) is an autosomal recessive neurodegenerative disorder caused by expansion of GAA repeats in intron 1 of the frataxin (FXN) gene, leading to significant decreased expression of frataxin, a mitochondrial iron-binding protein. We previously reported that syngeneic hematopoieti...

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Detalles Bibliográficos
Autores principales: Rocca, Celine J., Rainaldi, Joseph N., Sharma, Jay, Shi, Yanmeng, Haquang, Joseph H., Luebeck, Jens, Mali, Prashant, Cherqui, Stephanie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7240056/
https://www.ncbi.nlm.nih.gov/pubmed/32462051
http://dx.doi.org/10.1016/j.omtm.2020.04.018

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7240056/
https://www.ncbi.nlm.nih.gov/pubmed/32462051
http://dx.doi.org/10.1016/j.omtm.2020.04.018

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