ophthalmologyОфтальмологияOphthalmology in Russia1816-50952500-0845Ophthalmology10.18008/1816-5095-2025-2-244-250ophthalmology-2644Research ArticleОБЗОРЫREVIEWSИспользование генной терапии на основе AAV-векторов в лечении офтальмологических заболеваний (обзор литературы)The Use of AAV Vector-based Gene Therapy in the Treatment of Ophthalmic Diseases. Literature Reviewhttps://orcid.org/0009-0002-8666-2525БатыроваЗ. А.BatyrovaZ. A.

Батырова Залиха Расуловна, студентка 6‑го курса лечебного факультета

площадь Ленина, 1, Махачкала, 367000

Zalikha Rasulovna B. 6th year student 

Lenin sq., 1, Makhachkala, 367000

aspirant02@bk.ruhttps://orcid.org/0009-0001-4704-0777МуртазалиеваА. Ш.MurtazalievaA. Sh.

Муртазалиева Аминат Шамиловна, студентка 6‑го курса лечебного факультета

площадь Ленина, 1, Махачкала, 367000

Murtazalieva Aminat S. 6th year student 

Lenin sq., 1, Makhachkala, 367000

https://orcid.org/0009-0003-9909-2987РамазановаД. М.RamazanovaJ. M.

Рамазанова Джамиля Мавлудиновна, студентка 6‑го курса лечебного факультета

площадь Ленина, 1, Махачкала, 367000

Ramazanova Jamila M. 6th year student 

Lenin sq., 1, Makhachkala, 367000

https://orcid.org/0009-0005-3150-9664БатчаевА. С.-У.BatchaevA. S.‑U.

Батчаев Аслан Сеит‑Умарович, кандидат медицинских наук, доцент, и. о. заведующего кафедрой «Педиатрия»

ул. Ставропольская, 36, Черкесск, 369001

Batchaev Aslan S.‑U. PhD, Associate Professor, acting head of the Department of Pediatrics

Stavropolskaya str., 36, Cherkessk, 369001

ФГБОУ ВО «Дагестанский государственный медицинский университет» Министерства здравоохранения Российской ФедерацииРоссияDagestan State Medical UniversityRussian FederationФГБОУ ВО «Северо-Кавказская государственная академия»РоссияNorth Caucasus State AcademyRussian Federation202527062025222244250Copyright © Батырова З.А., Муртазалиева А.Ш., Рамазанова Д.М., Батчаев А.С., 20252025Батырова З.А., Муртазалиева А.Ш., Рамазанова Д.М., Батчаев А.С.Batyrova Z.A., Murtazalieva A.S., Ramazanova J.M., Batchaev A.S.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.ophthalmojournal.com/opht/article/view/2644

Данная обзорная статья посвящена изучению текущего состояния генной терапии с использованием аденоассоциированных вирусов (AAV) в лечении офтальмологических заболеваний.

Цель исследования — оценить эффективность и безопасность применения генной терапии в лечении наследственных и приобретенных заболеваний глаз, а также обсудить последние достижения в этой области. Проведен систематический обзор научной литературы из баз данных MEDLINE, PubMed, Киберленинка, Mendeley, Web of Science и EMBASE. Для поиска использовались ключевые термины и их комбинации: «генная терапия», «аденоассоциированный вирус», «офтальмология», «наследственные заболевания глаз», «возрастная макулярная дегенерация», «глаукома», «клинические испытания». В обзор включены клинические исследования, экспертные статьи и метаанализы, фокусирующиеся на применении AAV в терапии глазных патологий.

Результаты

Результаты. Исследования показывают, что генная терапия с использованием AAV представляет перспективное направление в лечении глазных заболеваний благодаря иммунной изоляции глаза и эффективности векторных систем. Однако вызванные AAV иммунные реакции остаются вызовом и требуют разработки специфических стратегий для их подавления. Подчеркивается значимость разработки менее иммуногенных капсидов и новых методов доставки векторов для повышения безопасности и эффективности терапии.

Заключение

Заключение. Генная терапия с использованием AAV открывает новые возможности для лечения наследственных и приобретенных заболеваний глаз. Несмотря на существующие проблемы, связанные с иммунными реакциями, современные достижения в области разработки векторов и методов доставки генетического материала предвещают высокие перспективы для более безопасных и эффективных терапевтических подходов.

Introduction

Introduction. This review article is devoted to the study of the current state of gene therapy using adeno-associated viruses (AAV) in the treatment of ophthalmic diseases.

Objective

Objective: The purpose: to evaluate the effectiveness and safety of using gene therapy in the treatment of hereditary and acquired eye diseases, as well as to discuss the latest achievements in this field. Materials and methods. A systematic review of scientific literature from the databases MEDLINE, PubMed, Cyberleninka, Mendeley, Web of Science and EMBASE was conducted. Key terms and their combinations were used for the search: “gene therapy”, “adeno-associated virus”, “ophthalmology”, “hereditary eye diseases”, “age-related macular degeneration”, “glaucoma”, “clinical trials”. The review includes clinical studies, expert articles, and meta-analyses focusing on the use of AAV in the treatment of ocular pathologies.

Results

Results. Research shows that gene therapy using AAV represents a promising direction in the treatment of eye diseases due to the immune isolation of the eye and the effectiveness of vector systems. However, AAV-induced immune responses remain a challenge and require the development of specific strategies to suppress them. The importance of developing less immunogenic capsids and new vector delivery methods to improve the safety and effectiveness of therapy is emphasized.

Conclusion

Conclusion. Gene therapy using AAV opens up new possibilities for the treatment of hereditary and acquired eye diseases. Despite the existing problems associated with immune responses, modern advances in the development of vectors and methods of delivering genetic material portend high prospects for safer and more effective therapeutic approaches.

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