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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...
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 1, 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 development for retinal diseases.

Felix F L Reichel1, Julia-Sophia Bellingrath2, Peter Kiraly3

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

Handbook of Clinical Neurology
|May 30, 2026
PubMed
Summary
This summary is machine-generated.

Gene therapy offers potential treatments for retinal diseases using gene augmentation and editing. Research focuses on delivery systems, challenges, and clinical applications for inherited retinal dystrophies.

Keywords:
Gene augmentationGene editingInherited retinal dystrophies (IRDs)Retinal gene therapyViral vectors

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Last Updated: Jun 1, 2026

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
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Published on: February 4, 2021

Area of Science:

  • Ophthalmology
  • Genetics
  • Biotechnology

Background:

  • Inherited and acquired retinal diseases represent a significant unmet medical need.
  • Gene therapy is emerging as a promising therapeutic modality for vision restoration.

Purpose of the Study:

  • To provide a comprehensive overview of current gene therapy strategies for retinal diseases.
  • To discuss challenges and clinical applications of retinal gene therapy.

Main Methods:

  • Review of gene augmentation and gene editing techniques.
  • Discussion of viral and nonviral delivery systems.
  • Analysis of challenges including gene identification, vector optimization, and immune responses.

Main Results:

  • Exploration of clinical applications in inherited retinal dystrophies.
  • Summary of recent clinical trial outcomes and endpoints.
  • Identification of key considerations for successful clinical translation.

Conclusions:

  • Retinal gene therapy is rapidly advancing, with ongoing research addressing key challenges.
  • Successful clinical translation requires careful consideration of delivery, safety, and efficacy.
  • Gene therapy holds significant promise for treating a spectrum of retinal conditions.