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

You might also read

Related Articles

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

Sort by
Same author

Structural dynamics insights into principles underlying the fitness of new broadly potent AAVs.

bioRxiv : the preprint server for biology·2026
Same author

Quantifying functional vision in a mouse model of oculocutaneous albinism type 1.

Scientific reports·2026
Same author

Synthesis and characterization of an injectable telechelic material for the epiretinal delivery of retinal gene therapies.

Journal of materials chemistry. B·2026
Same author

Interindividual variability in immune response to AAV ocular gene delivery across species impedes immunomonitoring.

JCI insight·2026
Same author

A mobile system for whole eye perfusion supporting retinal function and surgery.

Frontiers in bioengineering and biotechnology·2026
Same author

Ocular gene therapy mediated by AAV vectors: an overview of immunosuppressive treatments and immunomonitoring of patients involved in clinical trials.

Progress in retinal and eye research·2026

Related Experiment Video

Updated: Aug 5, 2025

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

Developing New Vectors for Retinal Gene Therapy.

Emilia A Zin1, Bilge E Ozturk2, Deniz Dalkara3

  • 1Sorbonne Université, INSERM, CNRS, Institut de la Vision, F-75012 Paris, France.

Cold Spring Harbor Perspectives in Medicine
|March 29, 2023
PubMed
Summary

Adeno-associated virus (AAV) vectors are key for in vivo gene delivery, especially in the eye. Enhancing AAV capsids is crucial for overcoming current limitations and achieving safe, efficient therapeutic gene delivery.

More Related Videos

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
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: Aug 5, 2025

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
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
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
  • Gene Therapy
  • Molecular Biology

Background:

  • Adeno-associated virus (AAV) vectors have been utilized for over 55 years for in vivo gene delivery.
  • Their application is particularly prominent in retinal gene therapy.
  • Advances in understanding AAV structure and biology have led to the development of engineered vectors.

Purpose of the Study:

  • To review recent advancements in engineering enhanced adeno-associated virus (AAV) capsids.
  • To highlight the persistent challenges in achieving safe and efficient therapeutic gene delivery using AAV vectors in the eye.

Main Methods:

  • Review of current literature on AAV capsid engineering.
  • Analysis of methods aimed at improving AAV vector performance for gene delivery.

Main Results:

  • Engineered AAV capsids show improved gene delivery capabilities.
  • Significant obstacles remain, including tropism, delivery efficiency, targeting, and payload capacity.

Conclusions:

  • Further improvements in AAV vector design are necessary for successful therapeutic applications in the eye.
  • Addressing current limitations is critical for maximizing therapeutic benefits while ensuring safety.