Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
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The pigmented epithelium, a bright partner against photoreceptor degeneration.Novel mobility test to assess functional vision in patients with inherited retinal dystrophies.Severe Loss of Tritan Color Discrimination in RPE65 Associated Leber Congenital Amaurosis.Genome Surgery and Gene Therapy in Retinal Disorders.Tapping the RNA world for therapeutics.Special Issue Introduction: Inherited Retinal Disease: Novel Candidate Genes, Genotype-Phenotype Correlations, and Inheritance Models.Presentation of TRPM1-Associated Congenital Stationary Night Blindness in Children.BEST1 gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure.Gene therapy and genome surgery in the retina.Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy.Progress in Gene Therapy to Prevent Retinal Ganglion Cell Loss in Glaucoma and Leber's Hereditary Optic Neuropathy.Early onset flecked retinal dystrophy associated with new compound heterozygous RPE65 variants.Prospect of retinal gene therapy following commercialization of voretigene neparvovec-rzyl for retinal dystrophy mediated by RPE65 mutation.Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA.A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular DystrophyRetained Plasticity and Substantial Recovery of Rod-Mediated Visual Acuity at the Visual Cortex in Blind Adult Mice with Retinal DystrophyTransfection by cationic gemini lipids and surfactantsEfficient Gene Transfer to Kidney Mesenchymal Cells Using a Synthetic Adeno-Associated Viral VectorCRISPR/Cas9 genome surgery for retinal diseasesHerpes Simplex Virus Vectors for Gene Transfer to the Central Nervous SystemPrecision medicine and clinical ophthalmology
P2860
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P2860
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
description
2017 nî lūn-bûn
@nan
2017年の論文
@ja
2017年論文
@yue
2017年論文
@zh-hant
2017年論文
@zh-hk
2017年論文
@zh-mo
2017年論文
@zh-tw
2017年论文
@wuu
2017年论文
@zh
2017年论文
@zh-cn
name
Efficacy and safety of voretig ...... ed, open-label, phase 3 trial.
@en
type
label
Efficacy and safety of voretig ...... ed, open-label, phase 3 trial.
@en
prefLabel
Efficacy and safety of voretig ...... ed, open-label, phase 3 trial.
@en
P2093
P2860
P50
P1433
P1476
Efficacy and safety of voretig ...... led, open-label, phase 3 trial
@en
P2093
Albert M Maguire
Amy Tillman
Bart P Leroy
Daniel C Chung
Dina Gewaily
Dominique Cross
F Parker Hudson
Francesca Simonelli
Hannah Reichert
J Fraser Wright
P2860
P304
P356
10.1016/S0140-6736(17)31868-8
P407
P577
2017-07-14T00:00:00Z