A study published in Ophthalmology last week showed hope for retinitis pigmentosa (RP) patients by using a gene editing technique called CRISPR.
What is CRISPR?
It’s a technology that allows researchers to edit DNA sequences which can alter gene function all non-invasively. Simply put, it can cut out the bad gene and insert a working one. This is great news for someone with a genetic disorder like RP.
Tell me about the study.
This study used CRISPR to treat autosomal dominant RP in mice. This is more challenging to treat because autosomal dominant RP patients inherit one defective gene and one normal gene so figuring out how to edit just one gene instead of both can be trickey. However, the team used a new strategy called “ablate and replace” that enabled them to maintain the working copy of the gene and edit only the defective one.
Did it work?
It did. The researchers used ERG’s to test the visual improvement and they found RP progression slowed in the treated mice.
The take home.
CRISPR technology is huge and the scientific community is freaking out over it’s potential application in genetic disorders. (via)
