AAV for Gene Therapy in Ocular Diseases: Progress and Prospects

Area of Science:

• Ophthalmology
• Molecular Biology
• Gene Therapy

Background:

• Gene therapy offers a promising one-time treatment for various eye diseases, particularly retinal conditions.
• Adeno-associated virus (AAV) is a leading viral vector for gene delivery due to its safety and broad tissue tropism.
• The eye's unique anatomy makes it an ideal target for AAV-mediated gene therapy, requiring small, stable doses.

Purpose of the Study:

• To review the biological functions and therapeutic applications of AAV in treating ocular diseases.
• To summarize the latest advancements in AAV-mediated gene editing and silencing strategies for genetic eye disorders.
• To discuss the progress in ocular optogenetics and the clinical translation of AAV gene therapy.

Main Methods:

• Comprehensive literature review of AAV biology, ocular gene therapy applications, and delivery routes.
• Systematic outline of AAV-mediated gene editing techniques, including base editing and prime editing.
• Analysis of AAV's role in optogenetic therapies and its application in animal models for ocular diseases.

Main Results:

• AAV vectors are effective for gene delivery in various ocular tissues, with numerous clinical trials ongoing.
• Gene editing and silencing strategies using AAV show potential for correcting genetic defects causing eye diseases.
• Optogenetic approaches with AAV offer new avenues for restoring vision in certain conditions.

Conclusions:

• AAV-mediated gene therapy is a rapidly advancing field with significant potential to revolutionize the management of ocular diseases.
• Continued research into gene editing, optogenetics, and overcoming clinical translation challenges is crucial for realizing AAV's full therapeutic capacity.
• This review highlights the progress and future directions of AAV gene therapy in ophthalmology.