Mutation-Independent Gene Therapies for Rod-Cone Dystrophies.

  title={Mutation-Independent Gene Therapies for Rod-Cone Dystrophies.},
  author={C{\'e}cile Fortuny and John G Flannery},
  journal={Advances in experimental medicine and biology},
The clinical success of gene replacement therapies in recent years has served as a proof of concept for the treatment of inherited retinal degenerations using adeno-associated virus (AAV) as viral vector. However, inherited retinal degenerative diseases showcase a broad genetic and mechanistic heterogeneity, challenging the development of mutation-specific… CONTINUE READING