Targeting ON-bipolar cells by AAV gene therapy stably reverses LRIT3-congenital stationary night blindness
Keiko Miyadera, Evelyn Santana, Karolina P. Roszak, Sommer Iffrig, Meike Visel, Simone Iwabe, Ryan F. Boyd, Joshua T. Bartoe, Yu Sato, Alexa P. Gray, Ana Ripolles‐Garcia, Valérie Dufour, Leah C. Byrne, John G. Flannery, William A. Beltran, Gustavo David Aguirre
This paper demonstrates that AAV gene therapy targeting ON-bipolar cells in a canine model of LRIT3-CSNB significantly improves night vision and restores LRIT3 function.
Proceedings of the National Academy of Sciences · Mar 22, 2022Peer-reviewedNeuroscience