Article
Exogenous photoreceptor-specific N-glycosylated PROM1 rescues retinal degeneration in patient and mouse models.
Molecular therapy : the journal of the American Society of Gene Therapy - 3 Jun 2026
Xu Ping, Guo Fuying, Wang Yuan, Chen Guifu, Song Xiaojing, Luo Bella Y, Zheng Dandan, Gao Guanjie, Yin Wenjing, Zhang Suai, Lahn Bruce T, Zhong Xiufeng
Abstract excerpt
Human prominin-1 (PROM1) is broadly expressed across multiple tissues. However, its pathogenic variants cause an exclusive retina-related disorder, PROM1-associated inherited retinal dystrophy (PROM1-IRD). The mechanistic basis underlying this tissue-specific vulnerability remains unclear, and no approved targeted therapy is available. Here, we used urine cells, human induced pluripotent stem cells (hiPSCs),...
Topics
- Animals
- Humans
- Mice
- AC133 Antigen
- Retinal Degeneration
- Disease Models, Animal
- Glycosylation
- Dependovirus
- Genetic Vectors
- Induced Pluripotent Stem Cells