scholarly article | Q13442814 |
P50 | author | Katherine A. High | Q41502889 |
Stephen R Russell | Q44883012 | ||
Arlene Drack | Q56529441 | ||
Elliott H Sohn | Q58398731 | ||
Edwin M. Stone | Q37381855 | ||
Chris A. Johnson | Q37831129 | ||
P2093 | author name string | Dina 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 | |||
P2860 | cites work | Resolving the clinical acuity categories "hand motion" and "counting fingers" using the Freiburg Visual Acuity Test (FrACT). | Q40051827 |
Genetics and phenotypes of RPE65 mutations in inherited retinal degeneration. | Q40736925 | ||
Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy. | Q40787452 | ||
Gene therapy restores vision in a canine model of childhood blindness | Q43590733 | ||
Identification of the RPE65 protein in mammalian cone photoreceptors. | Q43974161 | ||
In utero gene therapy rescues vision in a murine model of congenital blindness | Q44753954 | ||
Lentiviral vectors containing a retinal pigment epithelium specific promoter for leber congenital amaurosis gene therapy. Lentiviral gene therapy for LCA. | Q45865415 | ||
Identification of factors that contribute to recombinant AAV2 particle aggregation and methods to prevent its occurrence during vector purification and formulation | Q46553714 | ||
Novel mobility test to assess functional vision in patients with inherited retinal dystrophies | Q48143937 | ||
Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle. | Q52532886 | ||
Visual acuity measurements | Q79797879 | ||
Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: an open-label phase 1b trial | Q87404711 | ||
Omics in Ophthalmology: Advances in Genomics and Precision Medicine for Leber Congenital Amaurosis and Age-Related Macular Degeneration | Q89054563 | ||
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial | Q24608049 | ||
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration | Q24627838 | ||
The human visual cortex responds to gene therapy-mediated recovery of retinal function | Q24629697 | ||
Safety and efficacy of gene transfer for Leber's congenital amaurosis | Q24634724 | ||
Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or leber congenital amaurosis | Q24685618 | ||
Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey | Q27329160 | ||
RPE65 is present in human green/red cones and promotes photopigment regeneration in an in vitro cone cell model | Q28255485 | ||
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial | Q28262431 | ||
Comprehensive genotyping reveals RPE65 as the most frequently mutated gene in Leber congenital amaurosis in Denmark | Q28270488 | ||
Effect of gene therapy on visual function in Leber's congenital amaurosis | Q28277981 | ||
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 | ||
Identification of RDH10, an All-trans Retinol Dehydrogenase, in Retinal Muller Cells | Q28568047 | ||
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer | Q33315634 | ||
A pilot study of an acupuncture protocol to improve visual function in retinitis pigmentosa patients | Q33601217 | ||
Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle. | Q34015881 | ||
Human photoreceptor topography | Q34034782 | ||
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 amaurosis | Q35836291 | ||
Plasticity of the human visual system after retinal gene therapy in patients with Leber's congenital amaurosis | Q36198173 | ||
Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement | Q36598421 | ||
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness | Q36825024 | ||
Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2 | Q36906324 | ||
Immune responses to AAV vectors: overcoming barriers to successful gene therapy | Q36983740 | ||
Transient transfection methods for clinical adeno-associated viral vector production | Q37479802 | ||
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 Retinoids | Q38574403 | ||
P433 | issue | 10097 | |
P407 | language of work or name | English | Q1860 |
P921 | main subject | phase III clinical trial | Q42824827 |
P304 | page(s) | 849-860 | |
P577 | publication date | 2017-07-14 | |
P1433 | published in | The Lancet | Q939416 |
P1476 | title | Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial | |
P478 | volume | 390 |
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