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Related Experiment Video

Updated: Jun 24, 2026

Subretinal Transplantation of Human Embryonic Stem Cell-Derived Retinal Tissue in a Feline Large Animal Model
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Subretinal Transplantation of Human Embryonic Stem Cell-Derived Retinal Tissue in a Feline Large Animal Model

Published on: August 5, 2021

Prospects for retinal gene replacement therapy.

Alexander J Smith1, James W Bainbridge, Robin R Ali

  • 1University College London Institute of Ophthalmology, Department of Genetics, 11-43 Bath Street, London EC1 V 9EL, UK. alexander.smith@ucl.ac.uk

Trends in Genetics : TIG
|March 24, 2009
PubMed
Summary
This summary is machine-generated.

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Gene therapy offers a promising new treatment for inherited retinal degeneration, including Leber congenital amaurosis (LCA). Early trials show safety and effectiveness, paving the way for treating more genetic vision loss conditions.

Area of Science:

  • Ophthalmology
  • Genetics
  • Molecular Biology

Background:

  • Inherited retinal degeneration (IRD) affects 1 in 3000 people in Western countries.
  • Vision loss in IRDs results from mutations in over 100 different genes.
  • Current treatments for IRDs are limited.

Purpose of the Study:

  • To discuss the potential of gene therapy for treating IRDs.
  • To identify gene defects most amenable to gene therapy interventions.
  • To build upon preliminary clinical trial findings for Leber congenital amaurosis (LCA).

Main Methods:

  • Review of genetic causes of IRDs.
  • Analysis of gene functions.
  • Evaluation of preliminary gene therapy clinical trial data for LCA.

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Last Updated: Jun 24, 2026

Subretinal Transplantation of Human Embryonic Stem Cell-Derived Retinal Tissue in a Feline Large Animal Model
07:43

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Published on: August 5, 2021

A Step by Step Protocol for Subretinal Surgery in Rabbits
12:31

A Step by Step Protocol for Subretinal Surgery in Rabbits

Published on: September 13, 2016

Development of a Refined Protocol for Trans-scleral Subretinal Transplantation of Human Retinal Pigment Epithelial Cells into Rat Eyes
12:45

Development of a Refined Protocol for Trans-scleral Subretinal Transplantation of Human Retinal Pigment Epithelial Cells into Rat Eyes

Published on: August 12, 2017

Main Results:

  • Over 100 genes implicated in IRDs have been identified.
  • Gene therapy for a form of LCA has demonstrated safety and efficacy in early trials.
  • Specific gene defects are better suited for gene therapy approaches.

Conclusions:

  • Gene therapy holds significant potential for treating various forms of IRD.
  • Further research and development are warranted to expand gene therapy applications for genetic vision loss.
  • Targeting specific gene defects is crucial for successful gene therapy outcomes in IRDs.