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CRISPR/Cas9 gene editing for curing sickle cell disease

Sickle cell disease (SCD) is the most common monogenic blood disorder marked by severe pain, end-organ damage, and early mortality. Treatment options for SCD remain very limited. There are only four FDA approved drugs to reduce acute complications. The only curative therapy for SCD is hematopoietic...

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Publicado en:Transfus Apher Sci
Autores principales: Park, So Hyun, Bao, Gang
Formato: Artigo
Lenguaje:Inglês
Publicado: 2021
Materias:
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC8049447/
https://ncbi.nlm.nih.gov/pubmed/33455878
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.transci.2021.103060
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