相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders
Marina Pavlou et al.
EMBO MOLECULAR MEDICINE (2021)
AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer
Helena Costa Verdera et al.
MOLECULAR THERAPY (2020)
Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR
Jasmina Cehajic-Kapetanovic et al.
NATURE MEDICINE (2020)
The Evolution of Retinal Gene Therapy: From Clinical Trials to Clinical Practice
Mark E. F. Pennesi et al.
OPHTHALMOLOGY (2020)
Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium
Debra A. Thompson et al.
TRANSLATIONAL VISION SCIENCE & TECHNOLOGY (2020)
Harmonization of Outcomes and Vision Endpoints in Vision Restoration Trials: Recommendations from the International HOVER Taskforce
Lauren N. Ayton et al.
TRANSLATIONAL VISION SCIENCE & TECHNOLOGY (2020)
Validation of a Vision-Guided Mobility Assessment for RPE65-Associated Retinal Dystrophy
Neruban Kumaran et al.
TRANSLATIONAL VISION SCIENCE & TECHNOLOGY (2020)
Focused Update on AAV-Based Gene Therapy Clinical Trials for Inherited Retinal Degeneration
Paula I. Fuller-Carter et al.
BIODRUGS (2020)
Retinal Dystrophies and the Road to Treatment: Clinical Requirements and Considerations
Mays Talib et al.
ASIA-PACIFIC JOURNAL OF OPHTHALMOLOGY (2020)
Gene therapy for visual loss: Opportunities and concerns
Jia Hui Lee et al.
PROGRESS IN RETINAL AND EYE RESEARCH (2019)
Luxturna: FDA documents reveal the value of a costly gene therapy
Jonathan J. Darrow
DRUG DISCOVERY TODAY (2019)
Gene Therapy Leaves a Vicious Cycle
Reena Goswami et al.
FRONTIERS IN ONCOLOGY (2019)
Gene therapy clinical trials worldwide to 2017: An update
Samantha L. Ginn et al.
JOURNAL OF GENE MEDICINE (2018)
Safety and Long-Term Efficacy of AAV4 Gene Therapy in Patients with &ITRPE65 &ITLeber Congenital Amaurosis
Guylene Le Meur et al.
MOLECULAR THERAPY (2018)
Non-syndromic retinitis pigmentosa
Sanne K. Verbakel et al.
PROGRESS IN RETINAL AND EYE RESEARCH (2018)
Special Issue Introduction: Inherited Retinal Disease: Novel Candidate Genes, Genotype-Phenotype Correlations, and Inheritance Models
Frans P. M. Cremers et al.
GENES (2018)
Voretigene neparvovec-rzyl for the treatment of biallelic RPE65 mutation-associated retinal dystrophy
Stephen Russell et al.
EXPERT OPINION ON ORPHAN DRUGS (2018)
Reading charts in ophthalmology
W. Radner
GRAEFES ARCHIVE FOR CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY (2017)
AAV Infection: Protection from Cancer
Arun Srivastava et al.
HUMAN GENE THERAPY (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
Stephen Russell et al.
LANCET (2017)
Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward
Jean Bennett
MOLECULAR THERAPY (2017)
Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations
William A. Beltran et al.
MOLECULAR THERAPY (2017)
Retinal Gene Therapy: Surgical Vector Delivery in the Translation to Clinical Trials
G. Alex Ochakovski et al.
FRONTIERS IN NEUROSCIENCE (2017)
Target 5000: Target Capture Sequencing for Inherited Retinal Degenerations
Adrian Dockery et al.
GENES (2017)
Leber congenital amaurosis/early-onset severe retinal dystrophy: clinical features, molecular genetics and therapeutic interventions
Neruban Kumaran et al.
BRITISH JOURNAL OF OPHTHALMOLOGY (2017)
Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy
Richard G. Weleber et al.
OPHTHALMOLOGY (2016)
ROBINS-I: a tool for assessing risk of bias in non-randomised studies of interventions
Jonathan A. C. Sterne et al.
BMJ-BRITISH MEDICAL JOURNAL (2016)
ROBINS-I: a tool for assessing risk of bias in non-randomised studies of interventions
Jonathan A. C. Sterne et al.
BMJ-BRITISH MEDICAL JOURNAL (2016)
Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa
Wen-Tao Deng et al.
HUMAN GENE THERAPY (2015)
Adeno-associated Vector Toxicity-To Be or Not to Be?
Hildegard Buening et al.
MOLECULAR THERAPY (2015)
Long-Term Effect of Gene Therapy on Leber's Congenital Amaurosis
J. W. B. Bainbridge et al.
NEW ENGLAND JOURNAL OF MEDICINE (2015)
Effective delivery of large genes to the retina by dual AAV vectors
Ivana Trapani et al.
EMBO MOLECULAR MEDICINE (2014)
Three-Year Follow-up after Unilateral Subretinal Delivery of Adeno-Associated Virus in Patients with Leber Congenital Amaurosis Type 2
Francesco Testa et al.
OPHTHALMOLOGY (2013)
Visual acuity testing. From the laboratory to the clinic
Ian L. Bailey et al.
VISION RESEARCH (2013)
Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years
Samuel G. Jacobson et al.
ARCHIVES OF OPHTHALMOLOGY (2012)
The Cochrane Collaboration's tool for assessing risk of bias in randomised trials
Julian P. T. Higgins et al.
BMJ-BRITISH MEDICAL JOURNAL (2011)
Avoidable waste in the production and reporting of research evidence
Iain Chalmers et al.
LANCET (2009)
The PRISMA statement for reporting systematic reviews and meta-analyses of studies that evaluate healthcare interventions: explanation and elaboration
Alessandro Liberati et al.
BMJ-BRITISH MEDICAL JOURNAL (2009)
AAV-mediated gene therapy for retinal disorders: from mouse to man
P. K. Buch et al.
GENE THERAPY (2008)
Leber congenital amaurosis - A model for efficient genetic testing of heterogeneous disorders: LXIV Edward Jackson memorial lecture
Edwin M. Stone
AMERICAN JOURNAL OF OPHTHALMOLOGY (2007)
Full-field stimulus testing (FST) to quantify visual perception in severely blind candidates for treatment trials
Alejandro J. Roman et al.
PHYSIOLOGICAL MEASUREMENT (2007)
Knowledge translation: Closing the evidence-to-practice gap
Eddy S. Lang et al.
ANNALS OF EMERGENCY MEDICINE (2007)
Visual acuities hand motion and counting fingers can be quantified with the Freiburg visual acuity test
K Schulze-Bonsel et al.
INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2006)
Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle
TM Redmond et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)
An overview of Leber congenital amaurosis: A model to understand human retinal development
RK Koenekoop
SURVEY OF OPHTHALMOLOGY (2004)