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Related Concept Videos

Gene Therapy00:59

Gene Therapy

28.0K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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The Retinoblastoma Gene01:20

The Retinoblastoma Gene

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Tumor suppressor genes are normal genes that can slow down cell division, repair DNA mistakes, or program the cells for apoptosis in case of irreparable damage. Hence, they play an essential role in preventing the proliferation of damaged cells.
The first-ever tumor suppressor gene called Rb was identified in retinoblastoma - a rare eye tumor in children. In inherited forms of the disease, a child inherits one defective copy of the Rb gene, which predisposes them to retinoblastoma. However,...
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The Retinoblastoma Gene01:20

The Retinoblastoma Gene

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Related Experiment Video

Updated: Mar 22, 2026

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
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Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

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On Retinal Gene Therapy.

M Dominik Fischer1

  • 1University Eye Hospital, Centre for Ophthalmology, University of Tübingen, Tübingen, Germany.

Ophthalmologica. Journal International D'Ophtalmologie. International Journal of Ophthalmology. Zeitschrift Fur Augenheilkunde
|April 28, 2016
PubMed
Summary
This summary is machine-generated.

Gene therapy offers a promising treatment for inherited retinal degeneration and blindness. Ongoing clinical trials show progress, addressing challenges for future therapeutic development.

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Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
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Area of Science:

  • Ophthalmology
  • Genetics
  • Molecular Biology

Background:

  • Inherited retinal diseases (IRDs) stem from genetic mutations, leading to degeneration and blindness.
  • Current treatments for IRDs are limited, necessitating novel therapeutic strategies.
  • Retinal gene therapy has emerged as a significant advancement for treating rare genetic eye disorders.

Discussion:

  • Translating basic science discoveries into clinical applications for retinal gene therapy.
  • Evaluating the efficacy and safety of current gene therapy clinical trials for retinal conditions.
  • Identifying and overcoming potential obstacles in the progression of retinal gene therapy.

Key Insights:

  • Gene therapy is a viable therapeutic paradigm for inherited retinal degeneration.
  • Clinical trials demonstrate progress in treating genetic blindness.
  • Addressing challenges is crucial for the future of retinal gene therapy.

Outlook:

  • Continued advancements in gene editing and delivery technologies.
  • Expanding the scope of gene therapy to a wider range of retinal disorders.
  • Potential for restoring vision in patients with previously untreatable conditions.