Genome editing for sickle cell disease: still time to correct?

Genome editing for sickle cell disease: still time to correct?

Sickle cell disease (SCD) is an inherited blood disorder, due to a single point mutation in the β-globin gene (HBB) leading to multisystemic manifestations and it affects millions of people worldwide. The monogenic nature of the disease and the availability of autologous hematopoietic stem cells (HS...

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Bibliographic Details
Main Authors: Giulia Ceglie, Marco Lecis, Gabriele Canciani, Mattia Algeri, Giacomo Frati
Format: Article
Language:English
Published: Frontiers Media S.A. 2023-11-01
Series:Frontiers in Pediatrics
Subjects:
sickle cell disease
gene editing
fetal hemoglobin reactivation
globin genes regulation
gene therapy
CRISPR/Cas9
Online Access:https://www.frontiersin.org/articles/10.3389/fped.2023.1249275/full

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https://www.frontiersin.org/articles/10.3389/fped.2023.1249275/full

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