phgenomicsФармакогенетика и фармакогеномикаPharmacogenetics and Pharmacogenomics2588-05272686-8849LLC "Izdatelstvo OKI"10.37489/2588-0527-2025-4-18-28JUMKTZphgenomics-343Research ArticleНОВЫЕ ТЕХНОЛОГИИNEW TECHNOLOGIESГенная терапия в борьбе с возрастной макулярной дегенерацией: новые перспективы восстановления зренияGene therapy for age-related macular degeneration: new prospects for vision restorationhttps://orcid.org/0000-0001-5081-414XМошетоваЛ. К.MoshetovaL. K.

Мошетова Лариса Константиновна — д. м. н., профессор, академик РАН, заведующий кафедрой офтальмологии

Larisa K. Moshetova — Dr. Sci. (Med.), Professor, Academician of the Russian Academy of Sciences, Head of the Department of Ophthalmology

https://orcid.org/0000-0001-7886-7702СошинаМ. М.SoshinaM. M.

Сошина Мария Михайловна — к. м. н., медицинский советник Медицинского департамента

Maria M. Soshina — Cand. Sci. (Med.), Medical advisor

greenmaha@yandex.ruФГБОУ ДПО «Российская медицинская академия непрерывного профессионального образования»РоссияRussian Medical Academy of Continuous Professional EducationRussian FederationАО «Р-Фарм»РоссияJSC "R-Pharm"Russian Federation202525122025041828Copyright © Мошетова Л.К., Сошина М.М., 20262026Мошетова Л.К., Сошина М.М.Moshetova L.K., Soshina M.M.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.pharmacogenetics-pharmacogenomics.ru/jour/article/view/343

Генная терапия представляет собой перспективное направление в лечении возрастной макулярной дегенерации (ВМД), направленное на преодоление ограничений традиционной анти-VEGF и антикомплементной терапии. В обзоре рассматриваются современные стратегии генной терапии, основанные на использовании аденоассоциированных вирусных векторов для доставки генов, кодирующих белки, ингибирующие ангиогенез и воспаление. Обсуждаются ключевые кандидаты в генные препараты для лечения влажной и сухой форм ВМД, такие как иксобероген соропарвовек, ABBV-RGX-314, 4D-150, JNJ-1887 и другие, а также их клинические испытания. Подчёркиваются преимущества генной терапии, включая снижение частоты инъекций и долговременный эффект, а также анализируются проблемы, связанные с безопасностью, иммунным ответом, методами доставки и доступностью лечения. Статья даёт всесторонний обзор текущих достижений и будущих перспектив генной терапии ВМД.

Gene therapy is a promising approach for the treatment of age-related macular degeneration (AMD), aimed at overcoming the limitations of conventional anti-VEGF and anti-complement therapies. This review examines modern gene therapy strategies based on the use of adeno-associated viral vectors to deliver genes encoding proteins that inhibit angiogenesis and inflammation. Key gene therapy candidates for wet and dry AMD are discussed, including ixoberogene soroparvovec, ABBV-RGX-314, 4D-150, JNJ-1887, and others, along with their clinical trials. The advantages of gene therapy, such as reduced injection frequency and long-term efficacy, are highlighted, while challenges related to safety, immune response, delivery methods, and treatment accessibility are analyzed. The article provides a comprehensive overview of current advances and future prospects in gene therapy for AMD.

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The authors declare that there are no conflicts of interest present.