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Base and Prime Editing for Inherited Retinal Diseases: Delivery Platforms, Safety, Efficacy, and Translational
Haoliang Zhang1,2, Yuxuan Li3, Jiajie Li3
1Department of Ophthalmology, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200080, China.
Pharmaceutics
|November 27, 2025
Summary
Precision genome editing tools like base editing (BE) and prime editing (PE) offer new hope for treating inherited retinal diseases (IRDs). While promising, challenges in delivery and efficiency must be overcome for clinical success.
Area of Science:
- Ophthalmology
- Genetics
- Molecular Biology
Background:
- Inherited retinal diseases (IRDs) cause progressive vision loss.
- Current gene therapy (gene augmentation) has limitations for certain mutations and packaging capacity.
- Precision genome editing offers direct correction of pathogenic variants.
Purpose of the Study:
- Review progress in base editing (BE) and prime editing (PE) for IRDs.
- Highlight advancements in delivery systems (viral and non-viral).
- Summarize challenges and future perspectives for clinical translation.
Main Methods:
- Preclinical investigations in murine and non-human primate models.
- Evaluation of base editing and prime editing technologies.
- Analysis of delivery platform innovations and challenges.
Main Results:
- BE and PE demonstrate feasibility, accuracy, and preliminary safety in preclinical models.
- Significant challenges remain, including editing efficiency, off-target effects, and delivery to photoreceptors.
- Advancements in viral vector engineering and non-viral systems are underway.
Conclusions:
- BE and PE show potential for treating IRDs by directly correcting mutations.
- Overcoming delivery and efficiency hurdles is critical for clinical application.
- AI-driven strategies may accelerate the development of these therapies.
