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。Entering the Modern Era of Gene Therapy
Xavier M. Anguela et al.
ANNUAL REVIEW OF MEDICINE, VOL 70 (2019)
Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion
Jerry R. Mendell et al.
HUMAN GENE THERAPY (2019)
Impact of Age and Motor Function in a Phase 1/2A Study of Infants With SMA Type 1 Receiving Single-Dose Gene Replacement Therapy
Linda P. Lowes et al.
PEDIATRIC NEUROLOGY (2019)
Pre-market development times for biologic versus small-molecule drugs
Reed F. Beall et al.
NATURE BIOTECHNOLOGY (2019)
Gene Therapy
Katherine A. High et al.
NEW ENGLAND JOURNAL OF MEDICINE (2019)
From Clinical Trials to Clinical Practice: Practical Considerations for Gene Replacement Therapy in SMA Type 1
Samiah A. Al-Zaidy et al.
PEDIATRIC NEUROLOGY (2019)
Systemic gene transfer with rAAVrh74.MHCK7.SGCB increased β-sarcoglycan expression in patients with limb girdle muscular dystrophy type 2E
L. Rodino-Klapac et al.
NEUROMUSCULAR DISORDERS (2019)
AVXS-101 (Onasemnogene Abeparvovec) for SMA 1: Comparative Study with a Prospective Natural History Cohort
Samiah A. Al-Zaidy et al.
JOURNAL OF NEUROMUSCULAR DISEASES (2019)
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
Christian Hinderer et al.
HUMAN GENE THERAPY (2018)
Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy
Rachael A. Potter et al.
HUMAN GENE THERAPY (2018)
Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history
Harneet Arora et al.
MUSCLE & NERVE (2018)
Emerging Issues in AAV-Mediated In Vivo Gene Therapy
Pasqualina Colella et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)
Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids
Craig M. McDonald et al.
NEUROMUSCULAR DISORDERS (2018)
Comparison of Serum rAAV Serotype-Specific Antibodies in Patients with Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Inclusion Body Myositis, or GNE Myopathy
Deborah A. Zygmunt et al.
HUMAN GENE THERAPY (2017)
AAV: An Overview of Unanswered Questions
Kenneth I. Berns 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)
Systemic MV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice
Eric R. Pozsgai et al.
MOLECULAR THERAPY (2017)
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
J. R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report
Katy Benjamin et al.
VALUE IN HEALTH (2017)
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy
Caroline Le Guiner et al.
NATURE COMMUNICATIONS (2017)
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
Michael F. Naso et al.
BIODRUGS (2017)
Dystrophin contains multiple independent membrane-binding domains
Junling Zhao et al.
HUMAN MOLECULAR GENETICS (2016)
THE NEWCASTLE EXPERIENCE OF ANTI-MOG ASSOCIATED DISEASE
Clare Bolton et al.
JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2016)
DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study
Luca Bello et al.
NEUROLOGY (2016)
Clinical development of gene therapy: results and lessons from recent successes
Sandeep R. P. Kumar et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2016)
Gene therapy: progress and predictions
Mary Collins et al.
PROCEEDINGS OF THE ROYAL SOCIETY B-BIOLOGICAL SCIENCES (2015)
Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy
Balaji Balakrishnan et al.
CURRENT GENE THERAPY (2014)
Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery
L. G. Chicoine et al.
MOLECULAR THERAPY (2014)
Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes
Marika Pane et al.
PLOS ONE (2014)
Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy
Jerry R. Mendell et al.
MUSCLE & NERVE (2013)
Pre-existing Anti-Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy
Vedell Louis Jeune et al.
HUMAN GENE THERAPY METHODS (2013)
Evidence-Based Path to Newborn Screening for Duchenne Muscular Dystrophy
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2012)
Gene therapy for Duchenne muscular dystrophy
Ingrid E. C. Verhaart et al.
CURRENT OPINION IN NEUROLOGY (2012)
rAAV-Mediated Tumorigenesis: Still Unresolved After an AAV Assault
Paul N. Valdmanis et al.
MOLECULAR THERAPY (2012)
Assessing the potential for AAV vector genotoxicity in a murine model
Hojun Li et al.
BLOOD (2011)
The Path From Bench to Bedside: Considerations Before Starting the Journey
Mason Wright Freeman et al.
JOURNAL OF INVESTIGATIVE MEDICINE (2011)
Biomechanics of the sarcolemma and costameres in single skeletal muscle fibers from normal and dystrophin-null mice
K. P. Garcia-Pelagio et al.
JOURNAL OF MUSCLE RESEARCH AND CELL MOTILITY (2011)
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
Sebahattin Cirak et al.
LANCET (2011)
The Natural History of Cardiac and Pulmonary Function Decline in Patients With Duchenne Muscular Dystrophy
Rolando Roberto et al.
SPINE (2011)
Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors
Sylvie Boutin et al.
HUMAN GENE THERAPY (2010)
Eccentric contractions lead to myofibrillar dysfunction in muscular dystrophy
Bert Blaauw et al.
JOURNAL OF APPLIED PHYSIOLOGY (2010)
Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management
Katharine Bushby et al.
LANCET NEUROLOGY (2010)
Brief Report: Dystrophin Immunity in Duchenne's Muscular Dystrophy.
Jerry R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2010)
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
Steven J. Howe et al.
JOURNAL OF CLINICAL INVESTIGATION (2008)
Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
Carmela Zincarelli et al.
MOLECULAR THERAPY (2008)
A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy
Louise R. Rodino-Klapac et al.
JOURNAL OF TRANSLATIONAL MEDICINE (2007)
Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle
Maja Z. Salva et al.
MOLECULAR THERAPY (2007)
Retroviral gene therapy: Safety issues and possible solutions
Y Yi et al.
CURRENT GENE THERAPY (2005)
Gene transfer vector biodistribution: pivotal safety studies in clinical gene therapy development
P Gonin et al.
GENE THERAPY (2004)
Clades of Adeno-associated viruses are widely disseminated in human tissues
GP Gao et al.
JOURNAL OF VIROLOGY (2004)
Number and spatial distribution of nuclei in the muscle fibres of normal mice studied in vivo
JC Bruusgaard et al.
JOURNAL OF PHYSIOLOGY-LONDON (2003)
Occurrence of leukaemia following gene therapy of X-linked SCID
DB Kohn et al.
NATURE REVIEWS CANCER (2003)
Episomal persistence of recombinant adenoviral vector Genomes during the cell cycle in vivo
A Ehrhardt et al.
JOURNAL OF VIROLOGY (2003)
Progress and problems with the use of viral vectors for gene therapy
CE Thomas et al.
NATURE REVIEWS GENETICS (2003)
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
S Hacein-Bey-Abina et al.
NEW ENGLAND JOURNAL OF MEDICINE (2003)
Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors
AK Zaiss et al.
JOURNAL OF VIROLOGY (2002)
Function and genetics of dystrophin and dystrophin-related proteins in muscle
DJ Blake et al.
PHYSIOLOGICAL REVIEWS (2002)
Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy
SQ Harper et al.
NATURE MEDICINE (2002)
分享论文
