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。Manufacturing and Regulatory Strategies for Clinical AAV2-hRPE65
J. Fraser Wright et al.
CURRENT GENE THERAPY (2012)
Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice
Alison R. Amenta et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2011)
Sustained Alpha-Sarcoglycan Gene Expression after Gene Transfer in Limb-Girdle Muscular Dystrophy, Type 2D
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2010)
Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers
Natee Jearawiriyapaisarn et al.
CARDIOVASCULAR RESEARCH (2010)
Comparative Cardiac Gene Delivery of Adeno-Associated Virus Serotypes 1-9 reveals that AAV6 Mediates the Most Efficient Transduction in Mouse Heart
Carmela Zincarelli et al.
CTS-CLINICAL AND TRANSLATIONAL SCIENCE (2010)
Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino
B. Wu et al.
GENE THERAPY (2010)
Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene
B. M. Koppanati et al.
GENE THERAPY (2010)
Effects of irradiating adult mdx mice before full-length dystrophin cDNA transfer on host anti-dystrophin immunity
S. Eghtesad et al.
GENE THERAPY (2010)
Read-Through Strategies for Suppression of Nonsense Mutations in Duchenne/Becker Muscular Dystrophy: Aminoglycosides and Ataluren (PTC124)
Richard S. Finkel
JOURNAL OF CHILD NEUROLOGY (2010)
Immune Responses to AAV in Canine Muscle Monitored by Cellular Assays and Noninvasive Imaging
Zejing Wang et al.
MOLECULAR THERAPY (2010)
Dystrophin Delivery to Muscles of mdx Mice Using Lentiviral Vectors Leads to Myogenic Progenitor Targeting and Stable Gene Expression
En Kimura et al.
MOLECULAR THERAPY (2010)
Persistent Expression of FLAG-tagged Micro-dystrophin in Nonhuman Primates Following Intramuscular and Vascular Delivery
Louise R. Rodino-Klapac et al.
MOLECULAR THERAPY (2010)
Brief Report: Dystrophin Immunity in Duchenne's Muscular Dystrophy.
Jerry R. Mendell et al.
NEW ENGLAND JOURNAL OF MEDICINE (2010)
The Polyproline Site in Hinge 2 Influences the Functional Capacity of Truncated Dystrophins
Glen B. Banks et al.
PLOS GENETICS (2010)
Efficacy of Systemic Morpholino Exon-Skipping in Duchenne Dystrophy Dogs
Toshifumi Yokota et al.
ANNALS OF NEUROLOGY (2009)
RhoA leads to up-regulation and relocalization of utrophin in muscle fibers
Cecile Gauthier-Rouviere et al.
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2009)
AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells
Federico Mingozzi et al.
BLOOD (2009)
Systemic delivery of AAV8 in utero results in gene expression in diaphragm and limb muscle: treatment implications for muscle disorders
B. M. Koppanati et al.
GENE THERAPY (2009)
Theoretic Applicability of Antisense-Mediated Exon Skipping for Duchenne Muscular Dystrophy Mutations
Annemieke Aartsma-Rus et al.
HUMAN MUTATION (2009)
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. (vol 8, pg 918, 2009)
M. Kinali et al.
LANCET NEUROLOGY (2009)
Evaluation of Vascular Delivery Methodologies to Enhance rAAV6-mediated Gene Transfer to Canine Striated Musculature
Paul Gregorevic et al.
MOLECULAR THERAPY (2009)
Laminin-111 protein therapy prevents muscle disease in the mdx mouse model for Duchenne muscular dystrophy
Jachinta E. Rooney et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)
Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
Mark L. Brantly et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)
Functional Substitution by TAT-Utrophin in Dystrophin-Deficient Mice
Kevin J. Sonnemann et al.
PLOS MEDICINE (2009)
Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
Maria Kinali et al.
LANCET NEUROLOGY (2009)
Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function
HaiFang Yin et al.
HUMAN MOLECULAR GENETICS (2008)
Targeting Artificial Transcription Factors to the Utrophin A Promoter EFFECTS ON DYSTROPHIC PATHOLOGY AND MUSCLE FUNCTION
Yifan Lu et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2008)
Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice
Haifang Yin et al.
MOLECULAR THERAPY (2008)
A Single Intravenous Injection of Adeno-associated Virus Serotype-9 Leads to Whole Body Skeletal Muscle Transduction in Dogs
Yongping Yue et al.
MOLECULAR THERAPY (2008)
Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice
Guy L. Odom et al.
MOLECULAR THERAPY (2008)
Recombinant adeno-associated virus transduction and integration
Brian R. Schultz et al.
MOLECULAR THERAPY (2008)
Codon and mRNA Sequence Optimization of Microdystrophin Transgenes Improves Expression and Physiological Outcome in Dystrophic mdx Mice Following AAV2/8 Gene Transfer
Helen Foster et al.
MOLECULAR THERAPY (2008)
Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice
Paul Gregorevic et al.
MOLECULAR THERAPY (2008)
Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
Carmela Zincarelli et al.
MOLECULAR THERAPY (2008)
Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle
Gabriela D. Ivanova et al.
NUCLEIC ACIDS RESEARCH (2008)
IRES-Mediated Translation of Utrophin A Is Enhanced by Glucocorticoid Treatment in Skeletal Muscle Cells
Pedro Miura et al.
PLOS ONE (2008)
Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer
Bo Wu et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)
Local dystrophin restoration with antisense oligonucleotide PRO051
Judith C. van Deutekom et al.
NEW ENGLAND JOURNAL OF MEDICINE (2007)
Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice
Rachid Benchaouir et al.
CELL STEM CELL (2007)
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver
Federico Mingozzi et al.
BLOOD (2007)
Immune responses to AAV in clinical trials
Federico Mingozzi et al.
CURRENT GENE THERAPY (2007)
Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products
K. Yuasa et al.
GENE THERAPY (2007)
Morpholino oligomer -: Mediated Exon skipping averts the onset of dystrophic pathology in the mdx mouse
Sue Fletcher et al.
MOLECULAR THERAPY (2007)
PTC124 targets genetic disorders caused by nonsense mutations
Ellen M. Welch et al.
NATURE (2007)
Unique integration profiles in a canine model of long-term repopulating cells transduced with gammaretrovirus, lentivirus, or foamy virus
Brian C. Beard et al.
HUMAN GENE THERAPY (2007)
CD8+ T-cell responses to adeno-associated virus capsid in humans
Federico Mingozzi et al.
NATURE MEDICINE (2007)
Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63 % of patients with Duchenne muscular dystrophy
Christophe Beroud et al.
HUMAN MUTATION (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)
Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy
Zejing Wang et al.
HUMAN GENE THERAPY (2007)
Comparative analysis of antisense oligonucleotide sequences for targeted skipping of Exon 51 during dystrophin Pre-mRNA splicing in human muscle
V. Arechavala-Gomeza et al.
HUMAN GENE THERAPY (2007)
Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs
Maurilio Sampaolesi et al.
NATURE (2006)
Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy
Haiyan Jiang et al.
BLOOD (2006)
rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice
Paul Gregorevic et al.
NATURE MEDICINE (2006)
Utrophin is a calpain substrate in muscle cells
Isabelle Courdier-Fruh et al.
MUSCLE & NERVE (2006)
Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model
MA Denti et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)
Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: how close are we?
P Miura et al.
TRENDS IN MOLECULAR MEDICINE (2006)
Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response
CS Manno et al.
NATURE MEDICINE (2006)
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
J Alter et al.
NATURE MEDICINE (2006)
Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucteotide
S Fletcher et al.
JOURNAL OF GENE MEDICINE (2006)
Efficient myocyte gene delivery with complete cardiac surgical isolation in situ
CR Bridges et al.
JOURNAL OF THORACIC AND CARDIOVASCULAR SURGERY (2005)
Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector
LT Su et al.
CIRCULATION (2005)
Stable transduction of myogenic cells with lentiviral vectors expressing a minidystrophin
S Li et al.
GENE THERAPY (2005)
Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart
Z Wang et al.
NATURE BIOTECHNOLOGY (2005)
Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles
QL Lu et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)
Transduction of satellite cells after prenatal intramuscular administration of lentiviral vectors
TC MacKenzie et al.
JOURNAL OF GENE MEDICINE (2005)
Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells
E Bachrach et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2004)
CTLA4Ig delivered by high-capacity adenoviral vector induces stable expression of dystrophin in mdx mouse muscle
Z Jiang et al.
GENE THERAPY (2004)
Sustained muscle expression of dystrophin from a high-capacity adenoviral vector with systemic gene transfer of T cell costimulatory blockade
ZL Jiang et al.
MOLECULAR THERAPY (2004)
Systemic delivery of genes to striated muscles using adeno-associated viral vectors
P Gregorevic et al.
NATURE MEDICINE (2004)
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping
A Goyenvalle et al.
SCIENCE (2004)
Clades of Adeno-associated viruses are widely disseminated in human tissues
GP Gao et al.
JOURNAL OF VIROLOGY (2004)
Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates
N Brunetti-Pierri et al.
HUMAN GENE THERAPY (2004)
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
S Hacein-Bey-Abina et al.
SCIENCE (2003)
Correction of the dystrophic phenotype by in vivo targeting of muscle progenitor cells
GP Kobinger et al.
HUMAN GENE THERAPY (2003)
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
SE Raper et al.
MOLECULAR GENETICS AND METABOLISM (2003)
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
QL Lu et al.
NATURE MEDICINE (2003)
Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle
BL Gebski et al.
HUMAN MOLECULAR GENETICS (2003)
Pharmacological strategies for muscular dystrophy
TS Khurana et al.
NATURE REVIEWS DRUG DISCOVERY (2003)
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
CS Manno et al.
BLOOD (2003)
Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product
K Yuasa et al.
GENE THERAPY (2002)
ADAM12 alleviates the skeletal muscle pathology in mdx dystrophic mice
P Kronqvist et al.
AMERICAN JOURNAL OF PATHOLOGY (2002)
Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Δ48-50 DMD cells
FG De Angelis et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2002)
Micro-dystrophin cDNA ameliorates dystrophic phenotypes when introduced into mdx mice as a transgene
M Sakamoto et al.
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2002)
Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy
SQ Harper et al.
NATURE MEDICINE (2002)
Immune evasion by muscle-specific gene expression in dystrophic muscle
D Hartigan-O'Connor et al.
MOLECULAR THERAPY (2001)
Non-toxic ubiquitous over-expression of utrophin in the mdx mouse
R Fisher et al.
NEUROMUSCULAR DISORDERS (2001)
Enhanced expression of the α7β1 integrin reduces muscular dystrophy and restores viability in dystrophic mice
DJ Burkin et al.
JOURNAL OF CELL BIOLOGY (2001)
Muscle-specific promoters may be necessary for adeno-associated virus-mediated gene transfer in the treatment of muscular dystrophies
L Cordier et al.
HUMAN GENE THERAPY (2001)
Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
B Wang et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2000)
Immune responses to dystrophin: implications for gene therapy of Duchenne muscular dystrophy
A Ferrer et al.
GENE THERAPY (2000)
Modulation of starling forces and muscle fiber maturity permits adenovirus-mediated gene transfer to adult dystrophic (mdx) mice by the intravascular route
WK Cho et al.
HUMAN GENE THERAPY (2000)