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.

The role of epigenetics in cancer heterogeneity

Epigenetics in the context of cancer is still poorly explored. In the last two decades it has become apparent that cancer cells within a tumour may have distinctive genetic and epigenetic features which can be affected by anticancer treatments negatively or positively. This article summarises present knowledge on the role of the epigenome in tumourigenesis and subsequent growth, as well as discussing the links between genetics and epigenetics in cancer.