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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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Bibliografische gegevens
Gepubliceerd in:Transfus Apher Sci
Hoofdauteurs: Park, So Hyun, Bao, Gang
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2021
Onderwerpen:
Online toegang: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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