Mutation-Independent Gene Therapies for Rod-Cone Dystrophies.

@article{Fortuny2018MutationIndependentGT,
  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},
  year={2018},
  volume={1074},
  pages={75-81}
}
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