Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies

Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies

Beta-hemoglobinopathies are the most common genetic disorders worldwide, caused by a wide spectrum of mutations in the β-globin locus, and associated with morbidity and early mortality in case of patient non-adherence to supportive treatment. Allogeneic transplantation of hematopoietic stem cells (a...

Full description

Bibliographic Details
Main Authors: Kiriaki Paschoudi, Evangelia Yannaki, Nikoletta Psatha
Format: Article
Language:English
Published: MDPI AG 2023-05-01
Series:International Journal of Molecular Sciences
Subjects:
genome editing
hemoglobinopathies
sickle cell disease
thalassemia
CRISPR/Cas9
base editing
Online Access:https://www.mdpi.com/1422-0067/24/11/9527

Similar Items

Similar Items