Precision BioSciences has dosed the first individual with their novel gene editing approach for individuals with Duchenne mutations between exons 45 and 55
Precision BioSciences has shared that the first participant in the FUNCTION_DMD clinical trial has received their investigational gene editing treatment, PBGENE-DMD. PBGENE-DMD delivers ARCUS nucleases via AAV9 to cut out exons 45-55 of the dystrophin gene, a therapeutic approach amenable to individuals with Duchenne with mutations in that region.
You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.
We use cookies to ensure that we give you the best experience on our website. By continuing to use this site you are agreeing to accept our use of cookies. For more information about cookies please visit our Privacy Policy page.