Related Experiment Video

Updated: Oct 22, 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

3.2K

The Ocular Gene Delivery Landscape

Bhubanananda Sahu1, Isha Chug1, Hemant Khanna1

  • 1Department of Ophthalmology & Visual Sciences, UMass Medical School, Worcester, MA 01655, USA.

Biomolecules
|August 27, 2021
PubMed

Insights

Gene therapy offers new hope for genetic eye diseases following FDA approval. Research explores various ocular delivery methods, highlighting the need for efficient routes for effective treatments.

Area of Science:

  • Ophthalmology
  • Genetics
  • Pharmacology

Background:

  • The eye is a key area for developing genetic disease therapies.
  • FDA approval of a gene therapy for congenital blindness has spurred further research.
  • Numerous studies are investigating gene therapies for various ocular conditions.

Purpose of the Study:

  • To review the history and significance of gene therapy in ophthalmology.
  • To analyze current ocular drug delivery methods for gene therapy.
  • To discuss the advantages and disadvantages of different delivery routes.

Main Methods:

  • Literature review of gene therapy history and ocular delivery systems.
  • Analysis of existing research on gene therapy for eye diseases.
  • Comparative assessment of various ocular drug delivery routes and vehicles.

Main Results:

  • Gene therapy holds significant promise for treating inherited eye conditions.
  • Different parts of the eye present unique challenges and opportunities for drug delivery.
  • The effectiveness, safety, and duration of gene therapy depend heavily on the delivery method.

Conclusions:

  • Efficient and targeted drug delivery is paramount for successful ocular gene therapy.
  • Ongoing research aims to optimize delivery vehicles and routes for enhanced therapeutic outcomes.
  • Understanding delivery system limitations is crucial for advancing gene therapy in ophthalmology.
Keywords:
eyegene therapyretina

More Related Videos

Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research10:10

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

Published on: November 2, 2018

9.5K
Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium06:48

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

Published on: August 7, 2015

21.6K

Related Concept Videos

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...
994
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...
896
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...
765
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...
26.2K
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,...
4.3K