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Updated: Sep 9, 2025

Author Spotlight: Advancing Vision Restoration - Stem Cell-Based Therapy for Retinal Diseases
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Published on: October 6, 2023

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RPE65- レチノパシーにおけるVoretigene Neparvovecの有効性と安全性:日本における第3相試験の結果

Kaoru Fujinami1,2,3,4,5,6,7, Kunihiko Akiyama2,7, Kazushige Tsunoda2,6,7

  • 1Laboratory of Visual Physiology, Division of Vision Research, National Institute of Sensory Organs, NHO Tokyo Medical Center, Tokyo, Japan.

Ophthalmology science
|September 5, 2025
PubMed
まとめ

Voretigene neparvovec (VN) 遺伝子治療は,RPE65網膜症の日本人の患者で視力と光感を改善しました. この第3相臨床試験では,この治療は1年以上にわたって好ましい安全性を示した.

キーワード:
電気生理学フルフィールド刺激試験遺伝子療法RPE65 についてボレティゲン neparvovec

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