Gene Therapy with a Promoter Targeting Both Rods and Cones Rescues Retinal Degeneration Caused by AIPL1 Mutations

por Sun, Xun, Pawlyk, Basil, Xu, Xiaoyun, Liu, Xiaoqing, Bulgakov, Oleg, Adamian, Michael, Sandberg, Michael A., Khani, Shahrokh C., Tan, Mei-Hong, Smith, Alexander J., Ali, Robin R., Li, Tiansen
Publicado 2009

Leber Congenital Amaurosis Associated with AIPL1: Challenges in Ascribing Disease Causation, Clinical Findings, and Implications for Gene Therapy

por Tan, Mei Hong, Mackay, Donna S., Cowing, Jill, Tran, Hoai Viet, Smith, Alexander J., Wright, Genevieve A., Dev-Borman, Arundhati, Henderson, Robert H., Moradi, Phillip, Russell-Eggitt, Isabelle, MacLaren, Robert E., Robson, Anthony G., Cheetham, Michael E., Thompson, Dorothy A., Webster, Andrew R., Michaelides, Michel, Ali, Robin R., Moore, Anthony T.
Publicado 2012