Gene therapy for ocular diseases.

  title={Gene therapy for ocular diseases.},
  author={Melissa M Liu and Jingsheng Tuo and C. -K. Chan},
  journal={The British journal of ophthalmology},
  volume={95 5},
The eye is an easily accessible, highly compartmentalised and immune-privileged organ that offers unique advantages as a gene therapy target. Significant advancements have been made in understanding the genetic pathogenesis of ocular diseases, and gene replacement and gene silencing have been implicated as potentially efficacious therapies. Recent improvements have been made in the safety and specificity of vector-based ocular gene transfer methods. Proof-of-concept for vector-based gene… CONTINUE READING


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