Microreviews in Cell and Molecular Biology: CRISPR-Cas9 as TDT and SDC treatment

Abstract

Monogenic diseases such as sickle cell and Beta thalassemia as diseases have affected millions across the globe. A study directed by the CRISPR therapeutics and Vertex Pharmaceuticals utilized the CRISPR-Cas9 technique to genetically edit out BCL11A erythroid enhancer gene locus present in sickle cell affected blood.