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Updated: Jan 31, 2026

High-throughput Screening for Protein-based Inheritance in S. cerevisiae
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Published on: August 8, 2017

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Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques

Galaxy Y Cho1,2,3, Kyle Bolo4, Karen Sophia Park1,2

  • 1Jonas Children's Vision Care and Bernard & Shirlee Brown Glaucoma Laboratory, New York, USA.

Molecular Diagnosis & Therapy
|December 21, 2018
PubMed

Insights

Gene therapy offers promising results for inherited retinal dystrophies, a leading cause of blindness. Ongoing clinical trials show potential for vision restoration, but long-term durability and inflammation require further study.

Area of Science:

  • Ophthalmology
  • Genetics
  • Molecular Biology

Background:

  • Inherited retinal dystrophies (IRDs) are a significant cause of global blindness.
  • Current treatments for IRDs are limited, necessitating novel therapeutic approaches.

Purpose of the Study:

  • To review the application of gene therapy for IRDs.
  • To update on recent clinical trial outcomes.
  • To discuss future directions in gene therapy and genome surgery for retinal diseases.

Main Methods:

  • Review of completed Phase I, II, and III clinical trials for gene therapy in IRDs.
  • Analysis of reported functional outcomes and safety profiles.
  • Discussion of emerging technologies like genome surgery.

Main Results:

  • Gene therapy trials demonstrate promise in improving functional vision and safety.
  • Durability of therapeutic effects and inflammatory responses remain areas for continued investigation.
  • Recent trials provide valuable data on the efficacy and tolerability of gene augmentation strategies.

Conclusions:

  • Gene therapy is a rapidly advancing field with significant potential for treating IRDs.
  • Further research is needed to optimize treatment protocols and address long-term challenges.
  • Gene therapy and genome surgery represent the future of inherited blindness treatment.

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