Kennedys Disease Community
Could genome editing be a cure?

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04-17-2016, 07:41 PM
Could genome editing be a cure?
Read this article today in our Sunday paper. Genome editing reverses defects The article says that with this new technique, doctors can "fix" the genetic defects that cause sickle cell anemia or cystic fibrosis. Why not KD?
04-18-2016, 01:40 PM
Ed Meyertholen
There was a study using Duchenne's Muscular Dytrophy mice which used CRISPR to alter the genes in adult mice and it relieved some of the symptoms. There is no obvious reason that it could not work on KD. That said, there are still kinks to work out but I would not be surprised to see this as a treatment in the future.
04-18-2016, 02:15 PM
Thanks for the information. I hope they start asking for volunteers soon.
04-21-2016, 10:57 AM
Ed Meyertholen

They are several years away from humans, I expect. There is, however, a new trial for Hungtington's Disease forming using iRNA techniques. The same company has tested similar treatments for KD in mice so I would suspect that this is more likely to be in humans in the near (still years, though) future.
04-21-2016, 03:04 PM
Everything always seems to be years away. Maybe our kids or grand-kids will benefit. Thanks.
10-13-2017, 12:16 PM
Dan B
A U.S. FDA advisory committee has recommended approval of a gene therapy for an inherited retinal disease that causes blindness. The trade name of the drug is to be Luxturna, and the common name is Voretigene neparvovec.

I do not know if this brings us any closer to a treatment for KD, but it does involve a drug therapy that "repairs" a genetic mutation. News reports say that the proposed drug would be administered by a single injection into each eye; it does not require a long or complex course of therapy.
10-13-2017, 02:40 PM
Dan, thanks for posting about Luxturna. Note though it is a gene therapy, ie they insert a gene to produce a needed protein. This is a little different then what we require which is gene editing as our problem is producing a mutant protein that causes trouble. In our case it is just a deletion, remove the excess repeats, but the amount removed may need to be customized to the individual for optimal results
10-13-2017, 05:04 PM
I'm holding out hope for the RNA editing uses of CRISPR rCAS9. They've had strong success with hereditary ALS, Huntingtons, and I believe KD in mouse models with this. Instead of altering DNA they are altering RNA which is potentially less complicated, and skirts the moral arguments surrounding genome editing. Its coming from research at UCSD.
10-13-2017, 10:01 PM
I also find the RNA editing promising. DNA editing is kind of scary if the editing technique isn't 100% accurate/foolproof.