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

28.1K
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...
28.1K
Gene Therapy00:59

Gene Therapy

4.8K
4.8K
Ophthalmic Drug Delivery Systems01:23

Ophthalmic Drug Delivery Systems

212
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...
212

You might also read

Related Articles

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

Sort by
Same author

Bi-allelic variants in CDK20 cause a severe ciliopathy with midline brain and facial anomalies.

American journal of human genetics·2026
Same author

Clinically relevant AAV8- <i>PEX1</i> gene therapy preserves retinal integrity and function long-term in a murine model of Zellweger spectrum disorder.

bioRxiv : the preprint server for biology·2026
Same author

Genetic Modulators of Disease Penetrance.

Annual review of biomedical data science·2026
Same author

Dynamics of intronic polyadenylation in the hematopoietic lineage and its regulation by DNA methylation.

Genome research·2026
Same author

Clinical Spectrum of Hereditary Hemorrhagic Telangiectasia: Data from the Comprehensive HHT Outcomes Registry of the US (CHORUS).

Blood·2026
Same author

Start Making Sense: Addressing Reported Ambiguity of Final Margin Status for Oral Cavity Cancer.

Head & neck·2026

Related Experiment Video

Updated: Mar 29, 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

3.8K

An Autogenously Regulated Expression System for Gene Therapeutic Ocular Applications.

Matthew A Sochor1,2, Vidyullatha Vasireddy1, Theodore G Drivas1

  • 1Center for Advanced Retinal and Ocular Therapeutics, F. M. Kirby Center for Molecular Ophthalmology, Philadelphia, PA 19104, USA.

Scientific Reports
|November 25, 2015
PubMed
Summary

Scientists developed a novel gene therapy tool, the autogenous transgene regulatory system (ARES), to control gene expression. This system, inducible by Isopropyl β-D-1-thiogalactopyranoside (IPTG), shows promise for treating genetic diseases.

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

22.4K
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

9.9K

Related Experiment Videos

Last Updated: Mar 29, 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

3.8K
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

22.4K
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

9.9K

Area of Science:

  • Molecular Biology
  • Gene Therapy
  • Biotechnology

Background:

  • Effective gene therapy requires precise control over transgene expression.
  • Existing methods for regulating transgenes in vivo can be limited.

Purpose of the Study:

  • To develop and validate a novel, inducible transgene regulatory system for controlling gene expression in various cell types and in vivo.
  • To assess the efficacy and reversibility of the autogenous transgene regulatory system (ARES) in the mouse retina.

Main Methods:

  • Development of the autogenous transgene regulatory system (ARES) based on the bacterial lac repressor.
  • Testing ARES in bacteria and eukaryotic cells.
  • In vivo studies using adeno-associated virus (AAV) vectors in mouse retina, with transgene expression regulated by Isopropyl β-D-1-thiogalactopyranoside (IPTG).

Main Results:

  • The ARES system demonstrated successful control of transgene expression in bacteria, eukaryotic cells, and mouse retina.
  • Luciferase expression in the mouse retina was reversibly controlled over three induction-repression cycles via oral IPTG administration.
  • IPTG, a non-pharmacologic molecule, showed no off-target effects in mammals.

Conclusions:

  • The autogenous transgene regulatory system (ARES) offers a robust and reversible method for controlling transgene expression.
  • Oral administration of IPTG for in vivo transgene induction presents a promising strategy for future gene therapy applications.
  • This system holds significant potential for treating inherited and acquired genetic diseases.