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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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 inserted. The...
Gene Therapy00:59

Gene Therapy

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 inserted. The...
Angle Closure Glaucoma: Treatment01:28

Angle Closure Glaucoma: Treatment

Angle-closure glaucoma, or closed-angle glaucoma, is an eye condition where the iris bulges out and blocks the iridocorneal angle, resulting in a buildup of aqueous humor and increased intraocular pressure. Immediate medical attention is necessary due to the sudden onset of symptoms. The treatment for angle-closure glaucoma includes short-term and long-term approaches. Short-term treatment involves using eye drops like pilocarpine to lower intraocular pressure by increasing aqueous humor...
Open Angle Glaucoma: Treatment01:27

Open Angle Glaucoma: Treatment

In open-angle glaucoma, the iridocorneal angle remains open, but the trabecular meshwork becomes stiff, slowing down the outflow of aqueous humor. This causes a buildup of aqueous humor in the anterior chamber, leading to a sudden increase in intraocular pressure. The treatment for open-angle glaucoma focuses on reducing the elevated intraocular pressure by either decreasing the secretion of aqueous humor or increasing its outflow.
Drugs such as carbonic anhydrase inhibitors, α2- and...
iPS Cell Differentiation01:22

iPS Cell Differentiation

The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
The Retinoblastoma Gene01:20

The Retinoblastoma Gene

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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Related Experiment Video

Updated: May 10, 2026

Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye
05:09

Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye

Published on: November 25, 2012

Gene therapy for blindness.

José-Alain Sahel1, Botond Roska

  • 1INSERM UMR_S 968, UPMC, University of Paris 06, Institut de la Vision, Paris, France. j.sahel@gmail.com

Annual Review of Neuroscience
|June 4, 2013
PubMed
Summary
This summary is machine-generated.

Ocular gene therapy offers a promising approach to restore vision for the visually impaired. Advances in retinal gene therapy, including gene replacement and optogenetics, are paving the way for treating blindness.

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Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
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Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System
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Last Updated: May 10, 2026

Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye
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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
  • Neuroscience

Background:

  • Restoring sight for the visually impaired is a significant unmet medical need.
  • Ocular gene therapy is a viable strategy due to the eye's suitability for gene delivery and the genetic basis of many blinding diseases.

Purpose of the Study:

  • To review the concepts and recent advances in gene therapy within the retina.
  • To explore traditional and novel gene therapy approaches for vision restoration.

Main Methods:

  • Review of traditional gene therapy methods like gene replacement and neuroprotection.
  • Discussion of emerging strategies such as optogenetic therapies for vision restoration.

Main Results:

  • Significant progress has been made in ocular gene therapy.
  • Proof-of-concept studies are evaluating the safety and efficacy of human gene therapies for vision restoration.

Conclusions:

  • Advances in retinal gene therapy hold potential for treating a range of blinding conditions.
  • Retinal gene therapy research may inform future gene therapies for other neurological disorders.