Repair of a Splicing Defect in Erythroid Cells from Patients with β-Thalassemia/HbE Disorder
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
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1. Gene Editing of the Endogenous Cryptic 3′ Splice Site Corrects the RNA Splicing Defect in the β654-Thalassemia Mouse Model;Human Gene Therapy;2024-08-13
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4. Genetic Manipulation Strategies for β-Thalassemia: A Review;Frontiers in Pediatrics;2022-06-15
5. Restoration of correct βIVS2-654-globin mRNA splicing and HbA production by engineered U7 snRNA in β-thalassaemia/HbE erythroid cells;Scientific Reports;2019-05-21
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