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

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...
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,...
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,...

You might also read

Related Articles

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

Sort by
Same author

Short Report: An Examination of Children's Autism Traits and Their Association With Family Experience.

Autism research : official journal of the International Society for Autism Research·2026
Same author

Spatial Transcriptomics Recontextualizes the Cellular Environment of Conjunctival Melanoma.

medRxiv : the preprint server for health sciences·2026
Same author

Effect of icosapent ethyl treatment on colorectal tissue marine omega-3 polyunsaturated fatty acid levels among patients with a history of adenoma: a prospective, single-arm clinical trial.

The American journal of clinical nutrition·2026
Same author

Corneal Innervation Research at a Crossroads: A Tool-Driven Roadmap for the Future.

Investigative ophthalmology & visual science·2026
Same author

WISDOM randomized trial comparing risk-based versus annual breast cancer screening: study cohort characteristics and design.

NPJ breast cancer·2026
Same author

Utility of a Multiplex Molecular Respiratory Pathogen Panel on Clinical Management of Children in the Pediatric Emergency Department.

The Journal of molecular diagnostics : JMD·2026

Related Experiment Video

Updated: Jun 21, 2026

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

Recent advances in ocular gene therapy.

Daniel C Chung1, Vivian Lee, Albert M Maguire

  • 1F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA. dcc2@mail.med.upenn.edu

Current Opinion in Ophthalmology
|August 12, 2009
PubMed
Summary

Ocular gene therapy shows promise for treating retinal diseases like Leber's congenital amaurosis. Human trials using adeno-associated virus gene replacement demonstrate safety and efficacy, improving vision in patients.

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

Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research
10:10

Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research

Published on: November 2, 2018

Related Experiment Videos

Last Updated: Jun 21, 2026

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

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

Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research
10:10

Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research

Published on: November 2, 2018

Area of Science:

  • Ophthalmology
  • Genetics
  • Molecular Biology

Background:

  • Ocular gene therapy has advanced significantly due to improved viral vector delivery.
  • Leber's congenital amaurosis is a genetic eye disease targeted by gene therapy.

Purpose of the Study:

  • To review advances in ocular gene therapy, focusing on viral vector delivery.
  • To assess the efficacy and safety of in-vivo gene therapy for Leber's congenital amaurosis in human trials.

Main Methods:

  • Adeno-associated virus-mediated gene replacement was used in human clinical trials.
  • Safety and efficacy were evaluated through subjective and objective measures of vision.

Main Results:

  • Human trials for retinal degeneration showed encouraging preliminary safety and efficacy.
  • Patients demonstrated increased light sensitivity and visual acuity.
  • Subjective vision improvements were supported by objective tests like pupillary light response.

Conclusions:

  • Ongoing clinical trials confirm the safety and efficacy of adeno-associated virus gene therapy for retinal diseases.
  • These findings lay the groundwork for treating other ocular conditions with gene therapy.