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Related Experiment Video

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RNA therapeutics for retinal diseases.

Michael C Gemayel1, Ashay D Bhatwadekar1, Thomas Ciulla1,2,3

  • 1Department of Ophthalmology, Eugene and Marilyn Glick Eye Institute, Indiana University, Indianapolis, IN, USA.

Expert Opinion on Biological Therapy
|December 14, 2020
PubMed
Summary

Noncoding RNA (ncRNA) is crucial for retinal function and disease. RNA-based therapies, including siRNA and ASOs, show promise for treating retinal diseases like AMD and DR.

Keywords:
Antisense oligonucleotidesRNA therapeuticsinherited retinal diseasemicroRNAnoncoding RNAshort hairpin RNAsmall-interfering RNAtranslational read-through inducing drugs

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Area of Science:

  • Ophthalmology
  • Molecular Biology
  • Genetics

Background:

  • Noncoding RNA (ncRNA) regulates critical retinal functions such as apoptosis, inflammation, and phototransduction.
  • Altered ncRNA levels are observed in various retinal diseases.
  • MicroRNA (miRNA) and small-interfering RNA (siRNA) are key ncRNAs involved in post-transcriptional gene regulation and RNA interference (RNAi).

Purpose of the Study:

  • To review the role of ncRNA in retinal diseases, including age-related macular degeneration (AMD) and diabetic retinopathy (DR).
  • To discuss the therapeutic potential of RNA-based treatments for retinal conditions.

Main Methods:

  • Review of current literature on ncRNA in retinal biology and disease.
  • Analysis of emerging RNA-based therapeutic strategies.

Main Results:

  • ncRNA, particularly miRNA, plays a significant role in the pathogenesis of AMD and DR by regulating angiogenesis, oxidative stress, and inflammation.
  • Several RNA-based therapies, including siRNA, shRNA, antisense oligonucleotides (ASOs), and translational read-through inducing drugs (TRIDs), are under development.
  • These therapies are often administered intravitreally, offering potential for pan-retinal effects.

Conclusions:

  • RNA-based therapeutics hold significant promise for treating a range of retinal diseases, including AMD, DR, and inherited retinal diseases (IRDs).
  • Specific RNA-based approaches target gene silencing, splicing correction, and protein expression modulation for therapeutic benefit.