First Ever USH1B Non Human Primate Model Created!
We are funding ground breaking Usher syndrome research
The work we have been helping to fund for the past two years is officially advancing our race towards finding a treatment for Usher syndrome type 1B. Martha Neuringer and her team at OHSU have accomplished the first ever of its kind. They genetically edited (using CRISPR-cas9 technology) a Non-Human-Primate embryo, implanted that embryo into a mature female primate and successfully birthed a genetically modified NHP with an inherited retinal disease. The targeted gene was MYO7A (Usher syndrome type1B) and 6 months after Gema’s birth, Martha Neuringer and her team have confirmed the presence of all three phenotypes associated with Usher syndrome type 1b: balance problems associated w/ a damaged vestibular system, profound bi-lateral hearing loss, and the early onset of retinal degeneration - affirming the successful creation of an USH1B NHP model.
This is a tremendous scientific and medical accomplishment within the gene editing world AND a game-changing milestone for the advancement of Usher syndrome treatments. NHP’s are the gold standard for testing therapies in animal models, and prior to this breakthrough, there has never been a reliable animal model to test promising therapies for Usher syndrome type 1B. Without models you cannot test and validate therapies for clinical trials. The more accurate the model the faster and more confident we can test and refine therapies for advancing into clinical trials.
This first ever animal model creation also goes far beyond treating Usher syndrome. The tools and protocols that Martha and her team have designed, tested and validated are replicable for many inherited retinal diseases and potentially beyond. Our Save Sight Now supporters helped to fund this work, and the results are real, tangible and life changing. Overview image below is from 2022 ARVO.
If you’d like to help us continue funding groundbreaking, life changing research, click on the donate button below.