Gene Editing Still Needs Improved Accuracy to Fix Genetic Diseases Inside Bodies

There has been a lot of progress with gene editing to precisely engineer the genome. We are close to starting to fix major gene-related diseases like sickle cell anemia. Preclinical studies using gene editing to tackle genetic and infectious diseases have highlighted the therapeutic potential of this technology. How close are we to using gene editing tools for the treatment of haematological (blood) disorders and the hurdles that need to be overcome to achieve clinical success.

There remain major barriers associated with the editing-based therapeutic treatment of blood disorders that must be addressed to advance clinical applications that rely on genome editing. Some of these challenges include :
1. the delivery of the editing machinery ex vivo (outside) and in vivo (inside the body)
2. the ability to preserve the stemness and achieve high levels of engraftment of HSCs in vivo
3. the identification and reduction in genome-wide off-target effects induced by the nucleases.