Ontology highlight
ABSTRACT:
SUBMITTER: Cho GY
PROVIDER: S-EPMC6380912 | biostudies-literature | 2019 Feb
REPOSITORIES: biostudies-literature
Cho Galaxy Y GY Bolo Kyle K Park Karen Sophia KS Sengillo Jesse D JD Tsang Stephen H SH
Molecular diagnosis & therapy 20190201 1
Inherited retinal dystrophies cause progressive vision loss and are major contributors to blindness worldwide. Advances in gene therapy have brought molecular approaches into the realm of clinical trials for these incurable illnesses. Select phase I, II and III trials are complete and provide some promise in terms of functional outcomes and safety, although questions do remain over the durability of their effects and the prevalence of inflammatory reactions. This article reviews gene therapy as ...[more]