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Gene Therapy

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 Therapy

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...

Open Angle Glaucoma: Treatment

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...

Angle Closure Glaucoma: Treatment

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...

Ophthalmic Drug Delivery Systems

Ophthalmic Drug Delivery Systems

Ophthalmic drug delivery faces major limitations due to poor absorption across the corneal membrane. This process is primarily driven by diffusion and is influenced by two main factors: the physicochemical properties of the drug and tear drainage. Most ophthalmic drugs, such as pilocarpine, epinephrine, atropine, and local anesthetics, are weak bases. They are typically formulated at an acidic pH to enhance chemical stability. However, this leads to high ionization, reducing their ability to...

Glaucoma: Overview

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...

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Related Experiment Video

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

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

Published on: March 16, 2022

Gene therapy for ocular diseases.

Melissa M Liu¹, Jingsheng Tuo, Chi-Chao Chan

¹Immunopathology Section, Laboratory of Immunology, NIH/NEI, Bethesda, MD 20895-1857, USA.

The British Journal of Ophthalmology

|August 25, 2010

Summary

This summary is machine-generated.

Ocular gene therapy shows promise for treating genetic eye diseases. Early clinical trials for Leber congenital amaurosis are reporting successes, advancing treatments for various retinal and non-retinal conditions.

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

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Area of Science:

Ophthalmology
Genetics
Molecular Biology

Background:

The eye's unique properties make it an ideal target for gene therapy.
Genetic eye diseases have well-understood pathways, enabling targeted therapeutic strategies.
Gene replacement and silencing are key approaches in ocular gene therapy.

Purpose of the Study:

To review current advancements in ocular gene therapy.
To discuss future prospects and novel methods in ocular gene transfer.
To highlight progress in treating both retinal and non-retinal ocular diseases.

Main Methods:

Vector-based gene transfer using adeno-associated virus (AAV) and lentivirus.
Development of enhanced vector performance and regulatory mechanisms.
Utilizing experimental models for various ocular diseases.

Main Results:

Proof-of-concept established for vector-based gene therapies in ocular disease models.
Phase 1 clinical trials for Leber congenital amaurosis show preliminary success.
Gene therapy prospects improved for retinitis pigmentosa, Stargardt disease, AMD, uveitis, and glaucoma.

Conclusions:

Ocular gene therapy is progressing rapidly, with early clinical successes.
Novel vector technologies are enhancing gene transfer safety and efficacy.
Gene therapy holds significant potential for treating a wide range of human eye diseases.