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Inherited Retinal Disease Therapies Targeting Precursor Messenger Ribonucleic Acid

Di Huang1,2,3, Sue Fletcher1,3, Steve D Wilton1,3

  • 1Molecular Therapy Laboratory, Murdoch University, Murdoch 6150, Australia.

Vision (Basel, Switzerland)
|November 20, 2019
PubMed

Insights

Inherited retinal diseases can be treated by targeting genetic defects with novel therapies. Antisense oligomers offer a promising approach to correct aberrant splicing in pre-mRNA for treating these vision-impairing genetic conditions.

Area of Science:

  • Genetics
  • Ophthalmology
  • Molecular Biology

Background:

  • Inherited retinal diseases are diverse genetic conditions causing vision loss.
  • Identifying causative genes and developing genetic therapies has advanced significantly.
  • Aberrant splicing mutations, common in inherited diseases, present therapeutic targets.

Purpose of the Study:

  • To review therapeutic approaches for inherited retinal diseases.
  • To focus on strategies correcting pre-mRNA splicing and exon selection.
  • To discuss the clinical translation of splicing-targeted therapeutics.

Main Methods:

  • Review of current literature on inherited retinal diseases.
  • Analysis of therapeutic strategies targeting genetic defects.
  • Discussion of antisense oligomer-mediated splice intervention.

Main Results:

  • Significant progress in identifying disease genes and developing therapies.
  • Demonstrated feasibility of antisense oligomer splice intervention in various studies.
  • Identification of splicing correction as a viable therapeutic strategy.

Conclusions:

  • Splicing-based therapies, including antisense oligomers, show promise for inherited retinal diseases.
  • Targeting pre-mRNA splicing offers a novel therapeutic avenue.
  • Clinical translation of these emerging therapies faces challenges but holds potential.
Keywords:
alternative splicingantisense oligonucleotidesinherited retinal dystrophypre-mRNA splicing processretinitis pigmentosasplicing correction

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