AAV-DJ-Mediated MYOC Silencing as a Gene Therapy Approach for Myocilin-Associated Glaucoma.
Wan Yue, Liu Xiangxiang, Yan Xuejing, Wu Shen, Teng Yufei, Han Luyi et al.
AAV-DJ-shMYOC gene therapy reduced mutant myocilin, preventing IOP elevation, preserving RGCs and visual function in young mice. It also lowered IOP in aged mice, showing potential for myocilin-related glaucoma by targeting the underlying proteinopathy.