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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Veröffentlicht in:	Transfusion and apheresis science 2021-02, Vol.60 (1), p.103060-103060, Article 103060
Hauptverfasser:	Park, So Hyun, Bao, Gang
Format:	Artikel
Sprache:	eng
Schlagworte:	Anemia, Sickle Cell - therapy CRISPR-Cas Systems - genetics CRISPR/Cas9 Gene editing Gene Editing - methods Humans Sickle cell disease
Online-Zugang:	Volltext
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