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siRNA Therapeutics in Ocular Diseases
Javier Moreno-Montañés1, Anne-Marie Bleau2, Tamara Martínez2
1Clínica Universidad de Navarra, Pamplona, Spain. jmoreno@unav.es.
Methods in Molecular Biology (Clifton, N.J.)
|April 30, 2021
Summary
RNAi therapeutics show promise for eye diseases, with novel delivery strategies overcoming ocular barriers. Research focuses on administration routes, modifications, and delivery systems for efficient gene silencing, with many candidates in development.
Area of Science:
- Ophthalmology
- Molecular Biology
- Pharmacology
Background:
- RNA interference (RNAi) therapeutics, specifically small interfering RNAs (siRNAs), are increasingly explored for treating ocular diseases.
- The eye presents unique challenges for siRNA delivery due to its complex structure, distinct anterior and posterior segment barriers, and clearance mechanisms.
Purpose of the Study:
- To summarize recent advancements in siRNA delivery for ocular disorders.
- To cover routes of administration, chemical modifications, and delivery systems for effective gene silencing in the eye.
- To discuss methods for siRNA detection and quantification in ocular tissues and associated safety concerns.
Main Methods:
- Review of current literature on RNAi therapeutics for ocular diseases.
- Analysis of various administration routes (e.g., topical, intravitreal).
- Evaluation of chemical modifications and delivery systems (e.g., nanoparticles, conjugates) enhancing siRNA stability and targeting.
Main Results:
- Novel strategies have been developed to overcome ocular drug barriers for efficient siRNA delivery.
- Diverse chemical modifications and advanced delivery systems are being investigated to improve siRNA efficacy and safety.
- Methods for siRNA detection and quantification in ocular tissues are crucial for therapeutic development.
Conclusions:
- Despite challenges, the field of RNAi therapeutics for ocular diseases is rapidly advancing.
- Numerous siRNA candidates are progressing through preclinical and clinical development for various eye disorders.
- Addressing safety and regulatory requirements is essential for translating these therapies into patient care.
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