Manufacturers: Precision BioSciences, Inc.
PBGENE-DMD employs two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene, aiming to restore a near full-length functional dystrophin protein that more closely resembles normal dystrophin than synthetic, truncated microdystrophin approaches.
Duchenne muscular dystrophy (DMD)
1 associated conditions
