The CRISPR/Cas9 genetic editing system has soaked up the media spotlight faster than anything not named Kardashian. But unlike that famous clan, recent research indicates that there may be good reason that the science paparazzi just can’t seem to get enough of CRISPR/Cas9. After making the leap from basic discovery to zebrafish to monkeys, a superstar MIT team reported that the CRISPR/Cas9 system has ‘cured’ it’s first human related disease in mice by correcting a disease-related mutation and creating a healthy phenotype. Here’s what went down:
- The teams model of choice was the Fah mutation in liver cells, it models the hereditary human disease known as tyrosinemia.
- A CRISPR/Cas9 payload was shot into these mutant cells via hydrodynamic injection.
- The treatment created wild-type expression of the protein in the previously mutant cells.
- While only causing wild-type expression in 1 out of 250 liver cells, these rescued cells went on to correct the body weight loss phenotype associated with this hereditary disorder.
While some are still skeptical about the low rescue rate, it’s hard to argue with a rescued phenotype and no apparent off-target effects, which have hampered the clinical potential of previous genetic editing techniques, like zinc-finger nucleases. The authors believe that their work is a landmark, as it is the first study to show that the CRISPR/Cas9 genetic editing system can be used in adult animals, while also highlighting the world of potential for correcting human genetic diseases.
Go and see what CRISPR/Cas9 can do for you in Nature Biotechnology, April 2014