Precision Medicine Trials in Retinal Degenerations

Area of Science:

• Ophthalmology and Genetics
• Molecular Therapy and Genome Editing

Background:

• Pharmacochemical interventions have evolved to gene-based molecular therapies.
• Monogenic orphan diseases, particularly rare inherited retinal dystrophies, have been early targets for genome editing.
• Gene therapy represents a significant advancement in treating complex genetic disorders.

Purpose of the Study:

• To review current, ongoing, and future gene therapy trials for specific inherited retinal diseases.
• To highlight the potential of genome editing technologies in ophthalmology.
• To discuss the emergence of advanced gene editing strategies like base and prime editing.

Main Methods:

• Review of recent, ongoing, and planned clinical trials for gene therapy in retinal diseases.
• Analysis of the application of genome editing technologies in treating inherited retinal dystrophies.
• Exploration of emerging gene editing techniques such as base editing and prime editing.

Main Results:

• Gene therapy trials are actively progressing for choroideremia, X-linked retinitis pigmentosa, Stargardt disease, and age-related macular degeneration.
• These trials demonstrate the potential to halt the progression of severe retinal diseases.
• The development of base editing and prime editing signifies a new frontier in precision medicine.

Conclusions:

• Gene therapy is a promising approach for treating inherited retinal dystrophies.
• Genome editing technologies are revolutionizing the treatment landscape for rare genetic eye diseases.
• Future strategies like base and prime editing herald a new era of personalized genetic medicine.