Progress in Gene Therapy for Rhodopsin Autosomal Dominant Retinitis Pigmentosa

Area of Science:

• Ophthalmology
• Genetics
• Gene Therapy

Background:

• Autosomal dominant retinitis pigmentosa (RP) is an inherited retinal degeneration.
• Mutations in the rhodopsin gene (RHO) are a common cause of dominant RP.
• Current treatments are limited, necessitating the development of novel therapeutic strategies.

Purpose of the Study:

• To review proof-of-concept gene therapy studies for rhodopsin-associated autosomal dominant RP (RHO-adRP).
• To summarize gene therapy approaches, reagents, and delivery strategies used in animal models over the past two decades.
• To highlight key findings from these preclinical studies.

Main Methods:

• Systematic review of gene therapy studies for RHO-adRP in animal models.
• Tabular compilation of gene silencing reagents (e.g., ASOs, shRNA).
• Categorization of gene delivery strategies (e.g., viral vectors, non-viral methods).

Main Results:

• Multiple gene therapy approaches have demonstrated proof-of-concept in preclinical RHO-adRP models.
• Various gene silencing reagents and delivery systems have been evaluated.
• Significant progress has been made in developing potential therapeutic interventions.

Conclusions:

• Gene therapy represents a promising therapeutic avenue for RHO-adRP.
• Continued research and optimization of delivery systems are crucial for clinical translation.
• Preclinical studies provide a foundation for future human clinical trials in gene therapy for RP.