老年性黄斑变性的基因治疗。

Q3 Medicine

International Ophthalmology Clinics Pub Date : 2025-07-01 Epub Date: 2025-07-02 DOI:10.1097/IIO.0000000000000567

Henry W Zhou, Leo A Kim

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引用次数: 0

摘要

基因治疗的最新进展和当前AMD治疗领域的显著特征导致了对将基因治疗方法应用于AMD的兴趣增加。这篇综述将讨论药物给药方法，病毒和非病毒传递载体，以及目前基因治疗湿性和干性AMD的试验。给药途径包括视网膜下、玻璃体内和脉络膜上入路。病毒载体包括腺病毒、慢病毒和腺相关病毒（AAV）载体。非病毒载体包括脂质纳米颗粒（LNP）和基于聚合物的载体。目前湿AMD的试验包括ADVM-022和RGX-314。目前干性AMD的试验包括GT-005和JNJ-1887。

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Gene Therapy in Age-related Macular Degeneration.

Recent advances in gene therapy and salient features of the current AMD therapeutic landscape have led to increased interest in applying gene therapy approaches to AMD. This review will discuss approaches to drug administration, viral and non-viral delivery vectors, and current trials in gene therapy for both wet and dry AMD. Drug administration routes include subretinal, intravitreal, and suprachoroidal approaches. Viral vectors include adenoviral, lentiviral, and adeno-associated viral (AAV) vectors. Non-viral vectors include lipid nanoparticle (LNP) and polymer-based vectors. Current trials in wet AMD include ADVM-022 and RGX-314. Current trials in dry AMD include GT-005 and JNJ-1887.

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来源期刊

International Ophthalmology Clinics Medicine-Ophthalmology

CiteScore

1.40

自引率

0.00%

发文量

期刊介绍： International Ophthalmology Clinics is a valuable resource for any medical professional seeking to stay informed and up-to-date regarding developments in this dynamic specialty. Each issue of this quarterly publication presents a comprehensive review of a single topic in a new or changing area of ophthalmology. The timely, tightly focused review articles found in this publication give ophthalmologists the opportunity to benefit from the knowledge of leading experts in this rapidly changing field.