Ontology highlight
ABSTRACT:
SUBMITTER: Yang T
PROVIDER: S-EPMC4700113 | biostudies-literature | 2015 Dec
REPOSITORIES: biostudies-literature
Yang Tingting T Justus Sally S Li Yao Y Tsang Stephen H SH
Molecular therapy : the journal of the American Society of Gene Therapy 20150921 12
A retinal pigmented epithelial (RPE) disorder, bestrophinopathy has recently been proven to be amenable to gene and cell-based therapies in preclinical models. RPE disorders and allied retinal degenerations exhibit significant genetic heterogeneity, and diverse mutations can result in similar disease phenotypes. Several RPE disorders have recently become targets for gene therapies in humans. The year 2011 brought a new advance in cell-based therapies, with the Food and Drug Administration approv ...[more]