PBGENE-DMD is a gene editing approach for individuals with variants between exons 45 to 55. This gene editing approach uses a novel endonuclease (genetic scissors) delivered by AAV9 to create a large inframe deletion between exons 45 and 55. This deletion results in a in-frame variant that is capable of producing a functional dystrophin protein. Unlike gene replacement strategies where a transgene is introdcued into muscle cells to make a protein (microdystrophin gene therapy) this approach makes permenant changes to an individuals DNA to enable protein production.
A Phase 1/2 study is actively recruiting
This program is sponsored by Precision Biosciences
For information regarding current or upcoming clinical trials please visit our Explore Clinical Trials Page.
