CRISPR opens door to new type of medicine—'genome surgery'

CRISPR opens door to new type of medicine—'genome surgery'

6 years ago
Anonymous $oIHRkISgaL

https://phys.org/news/2018-10-crispr-door-medicinegenome-surgery.html

If successful, the trials will inaugurate a new era of "genome surgery"—the precision targeting of genetic defects in the genome, using CRISPR-Cas9 customized to individual patients. Such "bespoke" therapies can benefit small groups of individuals or families with particular genetic defects that would never be addressed by large pharmaceutical companies.

Johnsen and his family, including his daughter Greta, are carriers of a rare disorder, Best disease, that afflicts perhaps a thousand people nationwide and leads to early vision loss and eventual blindness, much like the more common macular degeneration.