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Updated: Feb 14, 2026

Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research
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在RPE65视网膜退行症的年轻患者中,基因治疗结果

Kirk A J Stephenson1, Abrar K Alsalamah2, Anupreet Tumber1

  • 1Department of Ophthalmology and Vision Sciences, The Hospital for Sick Children, Toronto, ON, Canada.

Canadian journal of ophthalmology. Journal canadien d'ophtalmologie
|February 12, 2026
PubMed
概括

沃雷特基因pararvovec-rzyl (VN) 基因疗法可以在患有RPE65-LCA的年轻患者中安全地改善视网膜敏感性. 虽然发生了胆管缩,但功能结果仍然是积极的,表明VN VN.

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