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Updated: May 28, 2025

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Precise Gene Editing Technologies in Retinal Applications.

Mehri Ahmadian1, Iskalen Cansu Topcu Okan1, Gokce Uyanik1

  • 1Molecular Biology, Genetics And Bioengineering Program, Sabanci University, Istanbul, Turkey.

Advances in Experimental Medicine and Biology
|February 10, 2025
PubMed
Summary
This summary is machine-generated.

Gene therapy offers new hope for inherited retinal diseases (IRDs). Advanced gene editing tools like CRISPR, base editing, and prime editing show promise for treating IRDs by correcting genetic defects.

Keywords:
Base editingGene therapyInherited retinal diseasesPrime editingRNA editingRpe65

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Area of Science:

  • Ophthalmology
  • Genetics
  • Molecular Biology

Background:

  • Inherited retinal diseases (IRDs) are a significant cause of vision loss.
  • Gene therapy, exemplified by RPE65 gene replacement, has shown success in treating certain IRDs.
  • Recent advancements in gene editing offer new therapeutic strategies beyond simple gene replacement.

Purpose of the Study:

  • To review and summarize the latest advancements in genome editing technologies.
  • To evaluate the potential applications of these novel gene editing tools in treating IRDs.
  • To highlight prime editing, base editing, and RNA editing as emerging therapeutic modalities.

Main Methods:

  • Review of current literature on gene editing technologies.
  • Focus on CRISPR-Cas9, base editing, prime editing, and RNA editing systems.
  • Analysis of their applicability and potential for in vivo genetic modification in retinal diseases.

Main Results:

  • Gene editing technologies enable precise in vivo genetic modifications.
  • CRISPR-Cas9, base editing, and prime editing offer versatile tools for genetic correction.
  • RNA editing provides a post-transcriptional approach to correct mutations.

Conclusions:

  • Advanced gene editing techniques, including prime editing, base editing, and RNA editing, hold significant promise for treating IRDs.
  • These technologies offer precise genetic correction strategies for various mutations underlying retinal diseases.
  • Further research and development are crucial for translating these editing tools into effective clinical therapies for IRDs.