Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.

scientific article published on 13 July 2017

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. is …
instance of (P31):
scholarly articleQ13442814

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P356DOI10.1016/S0140-6736(17)31868-8
P932PMC publication ID5726391
P698PubMed publication ID28712537

P50authorKatherine A. HighQ41502889
Stephen R RussellQ44883012
Arlene DrackQ56529441
Elliott H SohnQ58398731
Edwin M. StoneQ37381855
Chris A. JohnsonQ37831129
P2093author name stringDina Gewaily
Bart P Leroy
Janet Wittes
Francesca Simonelli
Jean Bennett
Vinit B Mahajan
Daniel C Chung
Okan Elci
Julia A Haller
Albert M Maguire
J Fraser Wright
Xiaosong Zhu
Wanda Pfeifer
Leslie Raffini
Lindsey A George
F Parker Hudson
Zi-Fan Yu
Maria Davis
Laura Dingfield
Kathleen A Marshall
Dominique Cross
Sarah McCague
Michelle Weckmann
Katie Wachtel
Amy Tillman
Hannah Reichert
Jean Walshire
Jennifer A Wellman
Julie Pappas
Taylor L Kehoe
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Identification of factors that contribute to recombinant AAV2 particle aggregation and methods to prevent its occurrence during vector purification and formulationQ46553714
Novel mobility test to assess functional vision in patients with inherited retinal dystrophiesQ48143937
Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle.Q52532886
Visual acuity measurementsQ79797879
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Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administrationQ24627838
The human visual cortex responds to gene therapy-mediated recovery of retinal functionQ24629697
Safety and efficacy of gene transfer for Leber's congenital amaurosisQ24634724
Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or leber congenital amaurosisQ24685618
Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkeyQ27329160
RPE65 is present in human green/red cones and promotes photopigment regeneration in an in vitro cone cell modelQ28255485
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trialQ28262431
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Effect of gene therapy on visual function in Leber's congenital amaurosisQ28277981
Safety and Proof-of-Concept Study of Oral QLT091001 in Retinitis Pigmentosa Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)Q28551677
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Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transferQ33315634
A pilot study of an acupuncture protocol to improve visual function in retinitis pigmentosa patientsQ33601217
Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle.Q34015881
Human photoreceptor topographyQ34034782
Improvement and decline in vision with gene therapy in childhood blindness.Q34474831
Vector platforms for gene therapy of inherited retinopathies.Q34561531
Long-term effect of gene therapy on Leber's congenital amaurosisQ35836291
Plasticity of the human visual system after retinal gene therapy in patients with Leber's congenital amaurosisQ36198173
Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvementQ36598421
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindnessQ36825024
Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2Q36906324
Immune responses to AAV vectors: overcoming barriers to successful gene therapyQ36983740
Transient transfection methods for clinical adeno-associated viral vector productionQ37479802
Psychophysical assessment of low visual function in patients with retinal degenerative diseases (RDDs) with the Diagnosys full-field stimulus threshold (D-FST).Q37656641
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.Q37701838
Manufacturing and regulatory strategies for clinical AAV2-hRPE65.Q37780248
Cone Health and RetinoidsQ38574403
P433issue10097
P407language of work or nameEnglishQ1860
P921main subjectphase III clinical trialQ42824827
P304page(s)849-860
P577publication date2017-07-14
P1433published inThe LancetQ939416
P1476titleEfficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
P478volume390

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