Once only thought possible in the realm of science fiction, today, scientists are able to edit genes in human embryos using a technique that employs a Clustered, Regularly Interspaced, Short Palindromic Repeat (CRISPR) and a CRISPR associated protein (Cas)—typically Cas-9.1 For ease, this comment will refer to the CRISPR system, inclusive of the Cas protein, as CRISPR/Cas-9.
David Cyranoski, a well-respected Nature editor, remarked that CRISPR/Cas-9 technologies will trigger a “Sputnik 2.0.” Shockingly, China has already used CRISPR/Cas-9 in twin girls to remove part of a gene that is responsible for causing HIV.4 Similarly, CRISPR/Cas-9 has been used on human cancer cells, and it has the potential to be therapeutic for aggressive forms of lung cancer. CRISPR/Cas-9 also enables researchers to produce specific types of tissues by altering genes in pluripotent stem cells. Furthermore, researchers can use CRISPR/Cas-9 to replicate the genetic basis for various human diseases, which will provide unprecedented insight into otherwise enigmatic diseases. Ultimately, at this early stage, the budding uses of CRISPR/Cas-9 extend to sickle cell anemia, cystic fibrosis,
muscular dystrophy, cancer, eye diseases, and HIV.