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Subretinal Transplantation of Human Embryonic Stem Cell-Derived Retinal Tissue in a Feline Large Animal Model
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Novel Therapies for Inherited Retinal Dystrophies
1Vitreoretinal Associates, Gainesville, FL 32607, USA.
Journal of Clinical Medicine
|December 17, 2024
Summary
The first approved retinal gene therapy, voretigene neparvovec, offers a new treatment option for inherited retinal diseases. This landmark therapy targets specific genetic mutations causing vision loss.
Area of Science:
- Ophthalmology
- Genetics
- Molecular Biology
Background:
- Inherited retinal diseases (IRDs) represent a significant cause of irreversible blindness.
- Current treatments for IRDs are limited, with a high unmet medical need.
- The advent of gene therapy offers a promising avenue for treating the genetic basis of these conditions.

