Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Gene Therapy00:59

Gene Therapy

25.6K
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...
25.6K
Open Angle Glaucoma: Treatment01:27

Open Angle Glaucoma: Treatment

481
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...
481
Angle Closure Glaucoma: Treatment01:28

Angle Closure Glaucoma: Treatment

575
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...
575
Glaucoma: Overview01:25

Glaucoma: Overview

629
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...
629

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Development of In Vitro Potency Assays for Gene Therapy Products Targeting cGMP-Related Retinopathies.

Methods in molecular biology (Clifton, N.J.)·2026
Same author

Quantitative Comparison of Two Novel Swept-Source Optical Coherence Tomography Angiography Devices.

Diagnostics (Basel, Switzerland)·2026
Same author

Mechanisms of Gas-Induced Posterior Vitreous Detachment: A Look Behind the Bubble Using Optical Coherence Tomography in Prone Position.

Journal of clinical medicine·2026
Same author

Quantifying segmentation sensitivity in OCTA: Device-specific profiles across three commercial platforms.

PloS one·2026
Same author

Inter-expert agreement on epithelial thickness mapping for refractive surgery screening: variability highlights need for standardization.

Journal of cataract and refractive surgery·2026
Same author

High-Dose 8 mg Aflibercept for Neovascular Age-Related Macular Degeneration: Who Is Being Treated with This New Agent?

Life (Basel, Switzerland)·2025

Related Experiment Video

Updated: Jul 24, 2025

Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models
06:16

Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models

Published on: March 16, 2022

3.0K

[Gene therapy in ophthalmology].

Claudia S Priglinger1, Maximilian J Gerhardt2, Günther Rudolph2

  • 1Augenklinik, Ludwig-Maximilians-Universität München, Mathildenstr. 8, 80336, München, Deutschland. Claudia.Priglinger@med.uni-muenchen.de.

Die Ophthalmologie
|July 7, 2023
PubMed
Summary

Gene therapy for inherited retinal dystrophies, like voretigene neparvovec-rzyl, shows promise but faces challenges extending to other conditions. This review covers gene therapy principles, technologies, and clinical considerations.

Keywords:
Age-related macular degenerationGene delivery systemsLeber’s hereditary optic neuropathyRPE65Voretigene neparvovec-rzyl

More Related Videos

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

21.4K
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

33.4K

Related Experiment Videos

Last Updated: Jul 24, 2025

Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models
06:16

Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models

Published on: March 16, 2022

3.0K
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

21.4K
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

33.4K

Area of Science:

  • Ophthalmology
  • Genetics
  • Molecular Biology

Background:

  • Hereditary retinal dystrophies, particularly those caused by RPE65 gene mutations, are treatable with gene therapy.
  • Voretigene neparvovec-rzyl, an FDA-approved gene augmentation therapy, utilizes an adeno-associated virus vector to deliver a functional RPE65 gene copy to retinal pigment epithelial cells.

Approach:

  • This review examines current gene therapy principles and technologies for retinal diseases.
  • It discusses the application of gene augmentation and supplementation strategies.
  • The article explores challenges, limitations, and practical aspects of gene therapy implementation.

Key Points:

  • The success of RPE65 gene therapy has spurred interest in applying similar approaches to non-genetic diseases like age-related macular degeneration.
  • However, the efficacy of gene therapy principles may not be universally transferable to all retinal dystrophies.
  • Consideration of disease stage, patient expectations, and treatment success evaluation are crucial for clinical practice.

Conclusions:

  • Gene therapy offers a viable treatment for specific inherited retinal dystrophies.
  • Further research is needed to overcome challenges in extending gene therapy to broader applications and diverse retinal conditions.
  • A comprehensive understanding of gene therapy technologies and clinical considerations is essential for successful patient outcomes.