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 Experiment Video

Updated: Jun 11, 2026

Vibratome Sectioning Mouse Retina to Prepare Photoreceptor Cultures
11:22

Vibratome Sectioning Mouse Retina to Prepare Photoreceptor Cultures

Published on: December 22, 2014

Optimized AAV5-RPGR ORF15 Gene Therapy Rescues Photoreceptor Structure and Function in XLRP Mouse Model.

Xiang Chen1, Xintong Xu2, Shanshan Cao3

  • 1Senior Department of Ophthalmology, Chinese People's Liberation Army General Hospital & Chinese PLA Medical School.

American Journal of Ophthalmology
|June 9, 2026
PubMed
Summary

Related Concept Videos

You might also read

Related Articles

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

Sort by
Same author

Effect of Nutrient Enrichment on Alpha and Beta Diversity of Macroinvertebrate Community in a Boreal River of Northern China.

Biology·2026
Same author

JAK-STAT signaling pathway in cancer: from molecular mechanisms to clinical intervention.

Molecular cancer·2026
Same author

High-definition Transcranial Direct Current Stimulation Improves Cognitive Function in Patients with Cerebral Small Vessel Disease: An Open-label Pilot Study Informed by Cross-sectional Analysis.

Current neuropharmacology·2026
Same author

Chiral Brønsted acid-catalyzed kinetic resolution of radical additions.

Nature communications·2026
Same author

Imlunestrant with or without abemaciclib in advanced breast cancer: safety analyses from the EMBER-3 trial.

NPJ breast cancer·2026
Same author

Enantioselective Synthesis of 2-Oxabicyclo[2.1.1]hexanes and Bicyclo[2.1.1]hexanes via Catalytic Asymmetric Intramolecular Photocycloadditions.

Nature communications·2026
Same journal

Corneal sensitivity changes and nerve plexus abnormalities in noninfectious anterior uveitis.

American journal of ophthalmology·2026
Same journal

Role of Menopausal Hormone Therapy on Strabismus, Strabismus Surgery, and Reoperation Rates.

American journal of ophthalmology·2026
Same journal

Forging Ahead: The Need for Improved Representation in Academic Ophthalmology.

American journal of ophthalmology·2026
Same journal

Clinical Utility of Ultra-Widefield Swept-Source OCT for Intraocular Tumors: Comparison With Ultrasonography, SD-OCT, and MRI.

American journal of ophthalmology·2026
Same journal

Therapeutic Advances in Corneal Scar management: Topical Treatments, Mesenchymal Cell Therapy and Stromal Transplantation.

American journal of ophthalmology·2026
Same journal

Increased Risk for Ocular Surface Neoplasia in Recipients of Solid Organ Transplant.

American journal of ophthalmology·2026
See all related articles
This summary is machine-generated.

This study developed a novel gene therapy for X-linked retinitis pigmentosa (XLRP) using an optimized rAAV5-RPGR vector. The therapy successfully preserved photoreceptor structure and function in a mouse model, showing promise for treating XLRP.

Area of Science:

  • Ophthalmology
  • Genetics
  • Gene Therapy

Background:

  • X-linked retinitis pigmentosa (XLRP) is a severe inherited retinal disease caused by mutations in the RPGR gene.
  • The RPGR ORF15 sequence is challenging to clone due to its instability, hindering gene therapy development.
  • Current treatments for XLRP are limited, necessitating novel therapeutic strategies.

Purpose of the Study:

  • To develop and evaluate a recombinant adeno-associated virus serotype 5 (rAAV5) vector carrying an optimized human RPGR ORF15 transgene (rAAV5-RPGR).
  • To address the challenges associated with the unstable wild-type RPGR ORF15 sequence for potential XLRP treatment.
  • To assess the preclinical efficacy and safety of rAAV5-RPGR gene therapy in a mouse model of XLRP.

Main Methods:

  • Designed an optimized RPGR ORF15 sequence to overcome cloning difficulties and improve expression.
Keywords:
AAV5Gene therapyPreclinical studiesRetinal degenerationX-linked retinitis pigmentosa

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

Related Experiment Videos

Last Updated: Jun 11, 2026

Vibratome Sectioning Mouse Retina to Prepare Photoreceptor Cultures
11:22

Vibratome Sectioning Mouse Retina to Prepare Photoreceptor Cultures

Published on: December 22, 2014

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

  • Validated in vitro expression in HEK 293T and 661W cells.
  • Generated and characterized a complete Rpgr knockout (Rpgr-KO) mouse model.
  • Administered rAAV5-RPGR via subretinal injection at varying doses to Rpgr-KO mice.
  • Evaluated therapeutic effects on photoreceptor structure, function (electroretinography), and safety in mice and rabbits.

    • Main Results:

    • The optimized vector enhanced RPGR protein expression by 3.3-fold in vitro and eliminated truncated isoforms.
    • In Rpgr-KO mice, rAAV5-RPGR demonstrated dose-dependent, photoreceptor-specific transgene expression.
    • High-dose treatment significantly preserved outer nuclear layer thickness, reduced rhodopsin mislocalization, and partially restored retinal function.
    • Electroretinography showed significant improvements in scotopic a-wave and photopic b-wave amplitudes in treated mice.
    • No vector-related toxicity was observed in rabbits.

    Conclusions:

    • rAAV5-RPGR effectively delivered and expressed the optimized RPGR-ORF15 transgene.
    • The gene therapy significantly preserved photoreceptor structure and function in a severe XLRP mouse model.
    • The study provides preclinical proof-of-concept for RPGR-targeted gene replacement therapy for XLRP with a favorable safety profile.