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。Evidence-Based Path to Newborn Screening for Duchenne Muscular Dystrophy
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2012)
Human ES- and iPS-Derived Myogenic Progenitors Restore DYSTROPHIN and Improve Contractility upon Transplantation in Dystrophic Mice
Radbod Darabi et al.
CELL STEM CELL (2012)
Stem cell therapies for muscle disorders
Francesco S. Tedesco et al.
CURRENT OPINION IN NEUROLOGY (2012)
Progress in gene therapy of dystrophic heart disease
Y. Lai et al.
GENE THERAPY (2012)
Novel Mini-Dystrophin Gene Dual Adeno-Associated Virus Vectors Restore Neuronal Nitric Oxide Synthase Expression at the Sarcolemma
Yadong Zhang et al.
HUMAN GENE THERAPY (2012)
Read-through compound 13 restores dystrophin expression and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy
Refik Kayali et al.
HUMAN MOLECULAR GENETICS (2012)
Hsp72 preserves muscle function and slows progression of severe muscular dystrophy
Stefan M. Gehrig et al.
NATURE (2012)
Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency following AAV5 Gene Transfer
William E. Grose et al.
PLOS ONE (2012)
Transplantation of Genetically Corrected Human iPSC-Derived Progenitors in Mice with Limb-Girdle Muscular Dystrophy
Francesco Saverio Tedesco et al.
SCIENCE TRANSLATIONAL MEDICINE (2012)
Early Treatment With Lisinopril and Spironolactone Preserves Cardiac and Skeletal Muscle in Duchenne Muscular Dystrophy Mice
Jill A. Rafael-Fortney et al.
CIRCULATION (2011)
Mitigation of muscular dystrophy in mice by SERCA overexpression in skeletal muscle
Sanjeewa A. Goonasekera et al.
JOURNAL OF CLINICAL INVESTIGATION (2011)
Nitrosative stress elicited by nNOS mu delocalization inhibits muscle force in dystrophin-null mice
Dejia Li et al.
JOURNAL OF PATHOLOGY (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)
Gene Therapy of mdx Mice With Large Truncated Dystrophins Generated by Recombination Using rAAV6
Guy L. Odom et al.
MOLECULAR THERAPY (2011)
Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy
Nathalie M. Goemans et al.
NEW ENGLAND JOURNAL OF MEDICINE (2011)
Daily Treatment with SMTC1100, a Novel Small Molecule Utrophin Upregulator, Dramatically Reduces the Dystrophic Symptoms in the mdx Mouse
Jonathon M. Tinsley et al.
PLOS ONE (2011)
Drug Discovery for Duchenne Muscular Dystrophy via Utrophin Promoter Activation Screening
Catherine Moorwood et al.
PLOS ONE (2011)
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)
Long-Term Systemic Administration of Unconjugated Morpholino Oligomers for Therapeutic Expression of Dystrophin by Exon Skipping in Skeletal Muscle: Implications for Cardiac Muscle Integrity
Alberto Malerba et al.
NUCLEIC ACID THERAPEUTICS (2011)
Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer
William Lostal et al.
HUMAN MOLECULAR GENETICS (2010)
Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO
HaiFang Yin et al.
MOLECULAR THERAPY (2010)
Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping
Aurelie Goyenvalle 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)
A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy
Martin Krahn et al.
SCIENCE TRANSLATIONAL MEDICINE (2010)
Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice
Paul T. Martin et al.
AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY (2009)
Efficacy of Systemic Morpholino Exon-Skipping in Duchenne Dystrophy Dogs
Toshifumi Yokota et al.
ANNALS OF NEUROLOGY (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)
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)
L-arginine decreases inflammation and modulates the nuclear factor-κB/matrix metalloproteinase cascade in mdx muscle fibers
Karim Hnia et al.
AMERICAN JOURNAL OF PATHOLOGY (2008)
Local dystrophin restoration with antisense oligonucleotide PRO051
Judith C. van Deutekom et al.
NEW ENGLAND JOURNAL OF MEDICINE (2007)
PTC124 targets genetic disorders caused by nonsense mutations
Ellen M. Welch et al.
NATURE (2007)
Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers
Samit Hirawat et al.
JOURNAL OF CLINICAL PHARMACOLOGY (2007)
Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs
Maurilio Sampaolesi et al.
NATURE (2006)
Myogenic progenitor cells and skeletal myogenesis in vertebrates
Margaret Buckingham
CURRENT OPINION IN GENETICS & DEVELOPMENT (2006)
rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice
Paul Gregorevic et al.
NATURE MEDICINE (2006)
Left ventricular synchronization by biventricular pacing in Becker muscular dystrophy as assessed by tissue Doppler imaging
C Stöllberger et al.
HEART & LUNG (2005)
Bone marrow stromal cells generate muscle cells and repair muscle degeneration
M Dezawa et al.
SCIENCE (2005)
Dystrophin expression in myofibers of Duchenne muscular dystrophy patients following intramuscular injections of normal myogenic cells
D Skuk et al.
MOLECULAR THERAPY (2004)
Cellular uptake of antisense morpholino oligomers conjugated to arginine-rich peptides
HM Moulton et al.
BIOCONJUGATE CHEMISTRY (2004)
Heregulin ameliorates the dystrophic phenotype in mdx mice
TOB Krag et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2004)
TGFβ/BMP activate the smooth muscle/bone differentiation programs in mesoangioblasts
E Tagliafico et al.
JOURNAL OF CELL SCIENCE (2004)
Mesoangioblasts - vascular progenitors for extravascular mesodermal tissues
G Cossu et al.
CURRENT OPINION IN GENETICS & DEVELOPMENT (2003)
Side population cells from diverse adult tissues are capable of in vitro hematopoietic differentiation
A Asakura et al.
EXPERIMENTAL HEMATOLOGY (2002)
Adeno-associated virus vector-mediated minidystrophin gene therapy improves dystrophic muscle contractile function in mdx mice
J Watchko et al.
HUMAN GENE THERAPY (2002)
Identification of a novel population of muscle stem cells in mice: potential for muscle regeneration
ZQ Qu-Petersen et al.
JOURNAL OF CELL BIOLOGY (2002)
Becker muscular dystrophy-related cardiomyopathy: A favorable response to medical therapy
AH Doing et al.
JOURNAL OF HEART AND LUNG TRANSPLANTATION (2002)
Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy
SQ Harper et al.
NATURE MEDICINE (2002)
Diagnosis of Duchenne dystrophy by enhanced detection of small mutations
JR Mendell et al.
NEUROLOGY (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)
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)
分享论文
