Voretigene neparvovec-rzyl for the treatment of biallelic RPE65 mutation–associated retinal dystrophy

@article{Russell2018VoretigeneNF,
  title={Voretigene neparvovec-rzyl for the treatment of biallelic RPE65 mutation–associated retinal dystrophy},
  author={S. Russell and J. Bennett and A. Maguire and K. High},
  journal={Expert Opinion on Orphan Drugs},
  year={2018},
  volume={6},
  pages={457 - 464}
}
ABSTRACT Introduction: Inherited retinal dystrophies (IRDs) are the major cause of familial blindness in the Western world, and until recently, have been devoid of pharmacologic treatment options. Biallelic mutations in the RPE65 gene cause a progressive IRD characterized by decreased light sensitivity, constricted visual fields, and impaired visual acuity. Voretigene neparvovec-rzyl (VN), an adeno-associated viral vector gene therapy that delivers a normal copy of the RPE65 gene to retinal… Expand
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Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders
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