FundedChildren’s Hospital of Philadelphia (CHOP) is exploring an investigational base editing strategy using mRNA-LNP (messenger RNA loaded lipid nanoparticles) to activate the paternal UBE3A gene as a potential treatment for Angelman syndrome.
A therapeutic approach using mRNA delivered by LNP to disrupt the UBE3A-ATS and prevent the silencing of the paternal copy of the UBE3A gene, enabling UBE3A protein to be made.
Marco CarpenterFind information on Angelman syndrome, research, clinical trials, resources, and ways to get involved.
