LAUSR

The CRSPR/Cas 9 adaptive bacterial immune system, operationalized as the Cas9/guide RNA platform, has garnered considerable attention as a potentially transformative technology in the creation and delivery of novel gene therapies for genetic disease. Compared with earlier technologies, the system offers greater precision and ease of use and offers promise for both ex vivo manipulation of somatic cells and in vivo editing through viral vectors, as well as, possibly, germ line alteration through in vitro use in human embryos. Potential applications include the treatment of sickle cell disease, retinitis pigmentosa, and β thalassemia. These applications are notable for not only their previous intractability to treatment but also their potential to address some large health disparities with regard to ethnic variation in the target population. The first clinical trials for the use of CRSPR-based therapies, for cancer, were reported in China in 2016. Research has …