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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...
Glaucoma: Overview01:25

Glaucoma: Overview

Glaucoma is an eye condition characterized by increased intraocular pressure that damages the retina and optic nerve, leading to irreversible blindness if left untreated. The human eye has various components, including the cornea, iris, pupil, lens, and optic nerve. Aqueous humor is secreted by the epithelium of the ciliary body in the posterior chamber and flows through the trabecular meshwork and canal of Schlemm, maintaining normal intraocular pressure. The trabecular meshwork and the canal...
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.

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Related Experiment Video

Updated: Jun 6, 2026

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
06:48

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

Published on: August 7, 2015

Gene therapy for vision loss -- recent developments.

Knut Stieger1, Birgit Lorenz

  • 1Department of Ophthalmology, Justus-Liebig-University, Giessen, Germany. Knut.Stieger@uniklinikum-giessen.de

Discovery Medicine
|December 3, 2010
PubMed
Summary
This summary is machine-generated.

Adeno-associated virus (AAV) gene therapy shows promise for inherited retinal disorders, improving vision safely. This experimental treatment is being explored for conditions like achromatopsia and Leber congenital amaurosis.

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Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System
07:04

Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System

Published on: February 4, 2021

Area of Science:

  • Ophthalmology
  • Genetics
  • Molecular Biology

Background:

  • Inherited retinal disorders cause progressive vision loss.
  • Mutations in genes like RPE65 lead to photoreceptor dysfunction.
  • Current treatments are limited, necessitating novel therapeutic approaches.

Purpose of the Study:

  • To evaluate the safety and efficacy of adeno-associated virus (AAV) mediated gene therapy for inherited retinal diseases.
  • To explore AAV gene therapy applications in various retinal blinding disorders.
  • To investigate potential gene therapy strategies for retinitis pigmentosa (RP).

Main Methods:

  • AAV-based gene transfer to deliver therapeutic genes to retinal cells.
  • Clinical trials involving patients with RPE65 mutations.
  • Preclinical studies in canine and mouse models of retinal degeneration.
  • Development of combined gene knockdown and gene addition therapy for autosomal dominant RP.
  • Investigating AAV transfer of bacterial rhodopsin for RP with unknown mutations.

Main Results:

  • Successful improvement of photoreceptor function in RPE65-associated inherited retinal blindness.
  • Demonstrated safety of AAV gene therapy in over 30 patients.
  • Positive outcomes in canine models of achromatopsia and mouse models of Leber congenital amaurosis.
  • Development of promising gene therapy approaches for various forms of RP.

Conclusions:

  • AAV-mediated gene therapy is a safe and effective strategy for certain inherited retinal disorders.
  • The technology holds significant potential for treating a broader range of retinal blinding conditions.
  • Ongoing research is expanding gene therapy options for complex retinal diseases like RP.