Multiplex CRISPR/Cas9 genome editing in hematopoietic stem cells for fetal hemoglobin reinduction generates chromosomal translocations

Sickle cell disease and β-thalassemia are common monogenic disorders that cause significant morbidity and mortality globally. The only curative treatment currently is allogeneic hematopoietic stem cell transplantation, which is unavailable to many patients due to a lack of matched donors and carries...

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Detalles Bibliográficos
Autores principales: Samuelson, Clare, Radtke, Stefan, Zhu, Haiying, Llewellyn, Mallory, Fields, Emily, Cook, Savannah, Huang, Meei-Li W., Jerome, Keith R., Kiem, Hans-Peter, Humbert, Olivier
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8605315/
https://www.ncbi.nlm.nih.gov/pubmed/34853798
http://dx.doi.org/10.1016/j.omtm.2021.10.008

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