CRISPR-Cas9 genome engineering: Treating inherited retinal degeneration.

ABSTRACT: Gene correction is a valuable strategy for treating inherited retinal degenerative diseases, a major cause of irreversible blindness worldwide. Single gene defects cause the majority of these retinal dystrophies. Gene augmentation holds great promise if delivered early in the course of the disease, however, many patients carry mutations in genes too large to be packaged into adeno-associated viral vectors and some, when overexpressed via heterologous promoters, induce retinal toxicity. In addition to the aforementioned challenges, some patients have sustained significant photoreceptor cell loss at the time of diagnosis, rendering gene replacement therapy insufficient to treat the disease. These patients will require cell replacement to restore useful vision. Fortunately, the advent of indu

SUBMITTER: Burnight ER 

PROVIDER: S-EPMC8210531 | biostudies-literature | 2018 Jul

REPOSITORIES: biostudies-literature