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Updated: May 8, 2026

Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye
Published on: November 25, 2012
[New treatments of hereditary blindness]
Mette Bertelsen1, Thomas Rosenberg, Michael Larsen
1Øjenklinikken, Kennedy Centret, Gammel Landevej 7, Glostrup. mette.bertelsen@regionh.dk
Abstract:
Ongoing clinical trials are targeting several previously intractable hereditary causes of blindness of congenital, childhood or early adulthood onset, mainly in the optic nerve and retina. The intended stage of initiation of the new therapeutic approaches ranges from neonatal life and a structurally intact retinal tissue to adult life with a complete loss of photoreceptors. It must be assumed that some of the trials will succeed in producing new therapies and action must be taken to refine and accelerate diagnostics and to preserve therapeutic potential in blind people.
Insights
New clinical trials offer hope for hereditary blindness. Advances in diagnostics and therapies aim to preserve vision for patients with optic nerve and retinal conditions.
Area of Science:
- Ophthalmology
- Genetics
- Regenerative Medicine
Context:
- Hereditary blindness, affecting the optic nerve and retina, presents significant challenges.
- Congenital, childhood, and early adulthood onset forms are being targeted by novel therapies.
Purpose:
- To summarize the current landscape of clinical trials for intractable hereditary blindness.
- To highlight the diverse therapeutic strategies and target patient populations.
Summary:
- Clinical trials are advancing treatments for hereditary optic nerve and retinal diseases.
- Therapeutic approaches range from early intervention in neonates to late-stage photoreceptor loss in adults.
- Successful trials necessitate improved diagnostics and methods to preserve therapeutic potential.
Impact:
- These advancements hold promise for restoring or preserving vision in individuals with previously untreatable genetic eye conditions.
- Accelerated diagnostics and preserved therapeutic potential are crucial for maximizing treatment efficacy.
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