Current trends in gene therapy for retinal diseases (Review)

Area of Science:

• Ophthalmology
• Genetics
• Molecular Biology

Background:

• The eye, particularly the retina, is an ideal target for genetic therapy due to its immune privilege, accessibility, and non-proliferating cells.
• Inherited and degenerative retinal disorders represent a significant unmet medical need, with many currently considered intractable.

Purpose of the Study:

• To review the current research status of genetic manipulation techniques for visual impairment caused by retinal disorders.
• To highlight the potential of gene therapy in addressing previously untreatable blinding diseases.

Main Methods:

• Review of current scientific literature on gene therapy for retinal disorders.
• Discussion of genetic manipulation techniques and their application in treating inherited and degenerative eye conditions.

Main Results:

• Gene therapy has demonstrated promising results in preclinical and early clinical studies for various retinal conditions.
• The unique anatomical and physiological features of the eye facilitate gene delivery and sustained transgene expression.

Conclusions:

• Genetic manipulation offers a promising therapeutic avenue for inherited and degenerative retinal diseases.
• Ongoing research is crucial to address remaining questions regarding the long-term efficacy and safety of ocular gene therapy.