Voretigene Neparvovec-Rzyl

Last Updated: 04/28/2026

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Found 167 publications

Adeno-Associated Virus Gene Delivery to Hard-to-Transduce Tissues: Biological Barriers, Engineering Strategies, and Clinical Challenges.

Adeno-Associated Virus Gene Delivery to Hard-to-Transduce Tissues: Biological Barriers, Engineering Strategies, and Clinical Challenges.

Journal: Human gene therapy

Published: April 04, 2026

A heterozygous pathogenic RPE65 variant phenocopies a mitochondrial retinopathy.

A heterozygous pathogenic RPE65 variant phenocopies a mitochondrial retinopathy.

Journal: Ophthalmic genetics

Published: March 18, 2026

Vision Outcomes in Children Treated With Voretigene Neparvovec for RPE65-Associated Retinopathy.

Vision Outcomes in Children Treated With Voretigene Neparvovec for RPE65-Associated Retinopathy.

Journal: JAMA ophthalmology

Published: January 02, 2026

Clinical and pharmacovigilance safety evaluation of LUXTURNA® (voretigene neparvovec-rzyl).

Clinical and pharmacovigilance safety evaluation of LUXTURNA® (voretigene neparvovec-rzyl).

Journal: Cutaneous and ocular toxicology

Published: October 27, 2025

Gene Therapy for Inherited Retinal Disease: Current Strategies, Personalized Medicine, and Future Implications-A Comprehensive Review.

Gene Therapy for Inherited Retinal Disease: Current Strategies, Personalized Medicine, and Future Implications-A Comprehensive Review.

Journal: Journal of personalized medicine

Published: October 20, 2025

Mechanisms and Functions of Chromophore Regeneration in the Classical Visual Cycle: Implications for Retinal Disease Pathogenesis and Therapy.

Mechanisms and Functions of Chromophore Regeneration in the Classical Visual Cycle: Implications for Retinal Disease Pathogenesis and Therapy.

Journal: Biomolecules

Published: October 17, 2025

Biologics-device combinations: Enabling prolonged therapies in the posterior segment ocular disease.

Biologics-device combinations: Enabling prolonged therapies in the posterior segment ocular disease.

Journal: Advanced drug delivery reviews

Published: October 12, 2025

One down but many more to go: the state of gene therapy for inherited retinal disease.

One down but many more to go: the state of gene therapy for inherited retinal disease.

Journal: Regenerative medicine

Published: October 07, 2025

Gene therapy outcomes in young patients with RPE65-retinal degeneration.

Gene therapy outcomes in young patients with RPE65-retinal degeneration.

Journal: Canadian journal of ophthalmology. Journal canadien d'ophtalmologie

Published: October 03, 2025

SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye.

SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye.

Journal: Human gene therapy

Published: September 26, 2025

Retinal gene therapy using epiretinal AAV-containing fibrin hydrogel implants.

Retinal gene therapy using epiretinal AAV-containing fibrin hydrogel implants.

Journal: Science advances

Published: September 05, 2025

Nonclinical toxicity study duration in AAV gene therapy development: Evidence from industry survey supports adequacy of short-term assessments.

Nonclinical toxicity study duration in AAV gene therapy development: Evidence from industry survey supports adequacy of short-term assessments.

Journal: Molecular therapy. Methods & clinical development

Published: August 14, 2025

Showing 1-12 of 167

Last Updated: 04/28/2026