Gene therapy for inherited retinal and optic nerve degenerations.

@article{Moore2018GeneTF,
  title={Gene therapy for inherited retinal and optic nerve degenerations.},
  author={Nicholas A Moore and N{\'u}ria Morral and Thomas A. Ciulla and Peter Bracha},
  journal={Expert opinion on biological therapy},
  year={2018},
  volume={18 1},
  pages={
          37-49
        }
}
  • Nicholas A Moore, Núria Morral, +1 author Peter Bracha
  • Published in
    Expert opinion on biological…
    2018
  • Medicine
  • INTRODUCTION The eye is a target for investigational gene therapy due to the monogenic nature of many inherited retinal and optic nerve degenerations (IRD), its accessibility, tight blood-ocular barrier, the ability to non-invasively monitor for functional and anatomic outcomes, as well as its relative immune privileged state.Vectors currently used in IRD clinical trials include adeno-associated virus (AAV), small single-stranded DNA viruses, and lentivirus, RNA viruses of the retrovirus family… CONTINUE READING

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