Last Updated: 09/26/2026
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Found 23 publications
Intravitreal Delivery of rAAV2tYF-CB-hRS1 Vector for Gene Augmentation Therapy in Patients with X-Linked Retinoschisis: 1-Year Clinical Results.
Journal: Ophthalmology. Retina
Published: April 24, 2022
Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy.
Journal: Human gene therapy
Published: May 17, 2020
Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa.
Journal: Human gene therapy
Published: January 08, 2020
Prospective Evaluation of Patients With X-Linked Retinoschisis During 18 Months.
Journal: Investigative ophthalmology & visual science
Published: December 15, 2018
Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study.
Journal: Molecular therapy. Methods & clinical development
Published: November 13, 2018
Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice.
Journal: Human gene therapy. Clinical development
Published: October 04, 2018
Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy.
Journal: Human gene therapy
Published: June 06, 2018
Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-Mutant Dogs.
Journal: Human gene therapy. Clinical development
Published: October 13, 2017
Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep.
Journal: Human gene therapy. Clinical development
Published: May 09, 2017
Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: February 12, 2017
REPEATABILITY AND LONGITUDINAL ASSESSMENT OF FOVEAL CONE STRUCTURE IN CNGB3-ASSOCIATED ACHROMATOPSIA.
Journal: Retina (Philadelphia, Pa.)
Published: February 02, 2017
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.
5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: December 13, 2016
Last Updated: 09/26/2026