Premature Aging, Obesity, Brain Disorders: 3 FrontRunners In The CRISP-R Therapy Race

CRISPR is the ultimate child star in the biomedical universe. Just six years old, the gene editing prodigy is now the subject of multiple clinical trials that aim to push the lab tech into the real world. In 2017, a 44-year-old man received the first-ever dose of gene therapy—in the form of zinc-finger nucleases—that targeted a deficient gene in his liver. This type of gene therapy, called “in vivo” in scientist-speak, is markedly different than the most common type these days.

So far, the only gene therapies on the market are CAR-Ts: a procedure targeting blood cancer that extracts a person’s immune cells, genetically edits them within the lab to boost their cancer-killing power, and then infuses them back into the body.

In vivo gene therapy is far more intimate: rather than extracting a person’s cells, a gene editing mix is directly injected into a person, with the hope of performing molecular surgery with a single shot. CRISPR is now making that possibility very real. With dozens of efforts in the making, from premature aging to obesity and developmental brain disorders, here are the frontrunners beyond CRISPR-based cancer therapy to watch out for.

Source: https://singularityhub.com/