Genetic disruption of the KLF1 gene to overexpress gamma ‐globin gene using CRISPR/Cas9 system

ConclusionsThese findings support the induction of an indel mutation in the KLF1 gene leading to a null allele. As a result, the effect of KLF1 on the expression of BCL11A is decreased and its inhibitory effect on γ‐globin gene expression is removed. Application of CRISPR technology to induce indel in KLF1 gene in adult erythroid progenitors may provide a method to activate fetal hemoglobin expression in individuals with β‐thalassemia or sickle cell disease. This article is protected by copyright. All rights reserved.
Source: The Journal of Gene Medicine - Category: Genetics & Stem Cells Authors: Tags: RESEARCH ARTICLE Source Type: research