Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies.

@article{Hollander2010LightingAC,
  title={Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies.},
  author={Anneke I. den Hollander and Aaron K Black and Jean Bennett and Frans P. M. Cremers},
  journal={The Journal of clinical investigation},
  year={2010},
  volume={120 9},
  pages={3042-53}
}
Nonsyndromic recessive retinal dystrophies cause severe visual impairment due to the death of photoreceptor and retinal pigment epithelium cells. These diseases until recently have been considered to be incurable. Molecular genetic studies in the last two decades have revealed the underlying molecular causes in approximately two-thirds of patients. The mammalian eye has been at the forefront of therapeutic trials based on gene augmentation in humans with an early-onset nonsyndromic recessive… CONTINUE READING
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