welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- login
Crispr Halted Muscular Dystrophy In Dogs. Someday, It Might Cure Humans.
Today, our partner Dr. Eric Olson and his team at Exonics, a company CureDuchenne helped form, published data in the online journal, Science that shows an increase in dystrophin restoration of 92% in the heart of the canine’s. As the only Duchenne nonprofit organization to support Dr. Eric Olson’s work through Exonics, and to provide the seed funding necessary to accelerate his work in this company, we could not be more thrilled. This is a true testament to the power of venture philanthropy and I want to thank each and every donor and supporter who has believed in our work, and we are grateful to Dr. Olson for his dedication to Duchenne patients. Although we still have a long way to go before we confirm this therapy will be safe and effective in humans, this dramatic dystrophin restoration offers hope to all families affected by this disease.
rareRelated
-
CRISPR-SKIP: Programmable gene splicing with single base editorsCRISPR gene editing has revolutionized b...
-
Santhera’s Raxone® receives first positive EAMS scientific opinion from UK’s MHRA in Duchenne muscular ...The aim of the EAMS is to provide patien...
-
New Analyses of Translarna™ (ataluren) Data from ACT DMD Presented at the 12th Annual European Pediatric Neurology...PTC Therapeutics, Inc. today announced t...
-
Autologous Cell Therapy Approach for Duchenne Muscular Dystrophy using PiggyBac Transposons and MesoangioblastsDuchenne muscular dystrophy (DMD) is a l...
-
Therapeutic advances in muscular dystrophyThe muscular dystrophies comprise a hete...
-
Clinical trial of gene therapy for Duchenne muscular dystrophy underway at University of Florida HealthScreening has resumed in a clinical tria...
-
SGT-001 improves muscle function of dogs with Duchenne muscular dystrophy, studies showSolid Biosciences’ SGT-001 improved mu...