Voretigene Neparvovec-rzyl for Treatment of RPE65-Mediated Inherited Retinal Diseases: A Model for Ocular Gene Therapy Development

Thomas A. Ciulla,Rehan M. Hussain,Audina M. Berrocal,Aaron Nagiel

Voretigene Neparvovec-rzyl for Treatment of RPE65-Mediated Inherited Retinal Diseases: A Model for Ocular Gene Therapy Development

2020

Thomas A. Ciulla
Rehan M. Hussain
Audina M. Berrocal
Aaron Nagiel

ABSTRACTIntroduction: Over a decade of research and development culminated in the 2017 United States Food and Drug Administration (FDA) approval of voretigene neparvovec-rzyl (VN) for RPE65 mutatio...

Keywords:

Genetic enhancement
drug administration
RPE65
Immunology
Leber's congenital amaurosis
Medicine
Retinal
Retinitis pigmentosa
Adeno-associated virus
food and drug administration

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