CRISPR/Cas-mediated DNA base-editing in gene therapy targeting β-hemoglobinopathies

CRISPR-Cas-mediated DNA base-editing is a step up from the famous CRISPR/Cas9 genome editing platform. Base-editing is capable of modifying haematopoietic stem and progenitor cells (HSPCs). This approach is paramount when silencing the genetic regulators such as BCL11A which affects the expression of β- and γ-globin genes in β-hemoglobinopathies – sickle cell disease and β-thalassemia. High performance rates of the base-editors instill hope in everyday use of these techniques in the clinical setting.