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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...
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.

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Related Experiment Video

Updated: May 9, 2026

Adenoviral Gene Therapy for Diabetic Keratopathy: Effects on Wound Healing and Stem Cell Marker Expression in Human Organ-cultured Corneas and Limbal Epithelial Cells
11:13

Adenoviral Gene Therapy for Diabetic Keratopathy: Effects on Wound Healing and Stem Cell Marker Expression in Human Organ-cultured Corneas and Limbal Epithelial Cells

Published on: April 7, 2016

Corneal gene therapy: basic science and translational perspective.

Rajiv R Mohan1, Jason T Rodier, Ajay Sharma

  • 1Harry S. Truman Memorial Veterans' Hospital, Columbia, Missouri, USA. mohanr@health.missouri.edu

The Ocular Surface
|July 11, 2013
PubMed
Summary
This summary is machine-generated.

Gene therapy offers a promising approach to combat corneal blindness, the third leading cause of vision loss globally. This review explores advanced gene therapy vectors and techniques for treating ocular surface diseases.

Keywords:
adeno-associated virusadenoviruscorneacorneal diseases and dystrophiesgene therapylentivirusnanoparticlesretrovirus

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Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
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Corneal Tissue Engineering: An In Vitro Model of the Stromal-nerve Interactions of the Human Cornea
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Corneal Tissue Engineering: An In Vitro Model of the Stromal-nerve Interactions of the Human Cornea

Published on: January 24, 2018

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Last Updated: May 9, 2026

Adenoviral Gene Therapy for Diabetic Keratopathy: Effects on Wound Healing and Stem Cell Marker Expression in Human Organ-cultured Corneas and Limbal Epithelial Cells
11:13

Adenoviral Gene Therapy for Diabetic Keratopathy: Effects on Wound Healing and Stem Cell Marker Expression in Human Organ-cultured Corneas and Limbal Epithelial Cells

Published on: April 7, 2016

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

Corneal Tissue Engineering: An In Vitro Model of the Stromal-nerve Interactions of the Human Cornea
07:35

Corneal Tissue Engineering: An In Vitro Model of the Stromal-nerve Interactions of the Human Cornea

Published on: January 24, 2018

Area of Science:

  • Ophthalmology
  • Molecular Biology
  • Biotechnology

Background:

  • Corneal blindness is a significant global health issue, ranking as the third leading cause of vision impairment.
  • The cornea's unique properties, including immune privilege and accessibility, make it an ideal target for gene therapy.
  • Gene therapy presents a novel and evolving strategy for restoring vision in patients with corneal diseases.

Purpose of the Study:

  • To review the fundamental science behind gene therapy vectors.
  • To summarize the current advancements in gene therapy for various ocular surface disorders.
  • To highlight the potential of gene therapy in treating corneal blindness.

Main Methods:

  • Review of scientific literature on gene therapy vectors and ocular applications.
  • Analysis of current gene-based interventions for corneal diseases.
  • Discussion of vector efficacy, delivery techniques, and therapeutic gene expression.

Main Results:

  • Significant progress has been made in developing diverse and effective gene therapy vectors.
  • Various delivery techniques and approaches have been refined for ocular gene therapy.
  • The field is moving towards maximizing therapeutic gene expression, minimizing toxicity, and ensuring a high safety profile.

Conclusions:

  • Gene therapy holds substantial promise for treating corneal blindness and other ocular surface diseases.
  • Continued research into efficient vectors and safe delivery methods is crucial for clinical success.
  • The accessibility and immune-privileged nature of the cornea facilitate gene therapy interventions for vision restoration.