Truncated Complement Factor H Y402 Gene Therapy Cures C3 Glomerulonephritis
Lindsey A. Chew, Daniel Grigsby, C. Garren Hester, Joshua Amason, W. Kyle McPherson, Edward J. Flynn, Meike Visel, Christopher R. Starr, John G. Flannery, Tylor R. Lewis, Catherine Bowes Rickman
This study shows that AAV-delivered truncated CFH gene therapy can reverse C3G in mice and suggests its potential for treating AMD.
Molecular Therapy · Apr 24, 2025Peer-reviewedPathology