A Review: Gene Therapy for Inherited Retinal Diseases: Clinical Advances, Regulatory Milestones, and Emerging Therapeutic Frontiers
Year: 2025
Type: article
Abstract: Background: Inherited retinal diseases (IRDs) are a genetically heterogeneous group of progressive disorders and a leading cause of visual impairment worldwide. Advances in molecular genetics and vector engineering have enabled the development of targeted gene therapies, culminating in the clinical approval of voretigene neparvovec-rzyl for RPE65-associated retinal dystrophy. Methods: This review critically evaluates published literature and clinical trial data on gene therapy approaches for IRDs, including gene augmentation, genome editing, and RNA-based modulation. Delivery strategies, vector platforms, regulatory frameworks, and ethical implications are also examined to provide a comprehensive overview of the current landscape and future directions. Results: Approved and investigational therapies demonstrate encouraging outcomes in restoring or stabilizing visual function across a spectrum of IRDs. The clinical pipeline includes AAV-based therapies targeting RPGR, CHM, and RS1, with evolving approaches such as dual-vector delivery, CRISPR/Cas9 gene editing, and antisense oligonucleotide therapy. Challenges related to immunogenicity, vector capacity, manufacturing scale-up, and equitable access remain prominent. Conclusion: Gene therapy for IRDs has entered a pivotal phase of clinical translation. While technological innovation continues to broaden therapeutic reach, long-term success will depend on overcoming logistical, regulatory, and ethical barriers. Future efforts should emphasize scalable delivery, global accessibility, and mutation-specific personalization to achieve meaningful impact in patient care.
Authors: Mirza Mohammed Ismail Baig, Qiu Aowang, Sun Ruxu, Wang Ningyu, Jiao Ziyun, Zhu Ziyu, Jassim Subair, Zhang Weiwe
Language: English
Subfield: Molecular Biology
Domain: Life Sciences
Open Access status: bronze
OpenAlex ID: W6949626597
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