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

ConclusionsThe findings obtained in the present study 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 an indel in the KLF1 gene in adult erythroid progenitors may provide a method for activating fetal hemoglobin expression in individuals with β‐thalassemia or sickle cell disease.
Source: The Journal of Gene Medicine - Category: Genetics & Stem Cells Authors: Tags: RESEARCH ARTICLE Source Type: research