Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies (Q38079633)

28 August 2017

title

Repair or replace? Exploiting novel gene and cell therapy strategies for muscular dystrophies (English)

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28 August 2017

Sara Benedetti

1

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28 August 2017

Hidetoshi Hoshiya

series ordinal

2

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28 August 2017

Francesco Saverio Tedesco

series ordinal

3

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28 August 2017

language of work or name

English

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publication date

4 March 2013

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28 August 2017

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28 August 2017

volume

280

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28 August 2017

issue

17

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28 August 2017

page(s)

4263-4280

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Dystrophin and mutations: one gene, several proteins, multiple phenotypes

28 August 2017

cites work

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Emerging drugs for Duchenne muscular dystrophy

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Pharmacologically targeting the primary defect and downstream pathology in Duchenne muscular dystrophy

21 January 2018

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Histone deacetylase inhibitors in the treatment of muscular dystrophies: epigenetic drugs for genetic diseases.

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Pathophysiology and Therapy of Cardiac Dysfunction in Duchenne Muscular Dystrophy

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Progress in gene therapy of dystrophic heart disease

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Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

21 January 2018

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An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus

21 January 2018

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Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides

21 January 2018

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Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle.

21 January 2018

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Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

21 January 2018

1 reference

Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice

21 January 2018

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Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

21 January 2018

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Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

21 January 2018

1 reference

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping

21 January 2018

1 reference

Overview on DMD exon skipping

21 January 2018

1 reference

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

21 January 2018

1 reference

Systemic administration of PRO051 in Duchenne's muscular dystrophy

21 January 2018

1 reference

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

21 January 2018

1 reference

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 Delta 48-50 DMD cells

21 January 2018

1 reference

Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts

21 January 2018

1 reference

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

21 January 2018

1 reference

Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping.

21 January 2018

1 reference

Long-term benefit of adeno-associated virus/antisense-mediated exon skipping in dystrophic mice.

21 January 2018

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Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping

21 January 2018

1 reference

Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy

21 January 2018

1 reference

Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping

21 January 2018

1 reference

Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA.

21 January 2018

1 reference

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

21 January 2018

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RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1

21 January 2018

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Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping

21 January 2018

1 reference

Pathogenic exon-trapping by SVA retrotransposon and rescue in Fukuyama muscular dystrophy

21 January 2018

1 reference

Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124).

21 January 2018

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Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice

21 January 2018

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Premature stop codons involved in muscular dystrophies show a broad spectrum of readthrough efficiencies in response to gentamicin treatment.

21 January 2018

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Drug-induced readthrough of premature stop codons leads to the stabilization of laminin alpha2 chain mRNA in CMD myotubes

21 January 2018

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PTC124 targets genetic disorders caused by nonsense mutations

21 January 2018

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Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers

21 January 2018

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Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression

21 January 2018

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Homing endonuclease structure and function

21 January 2018

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A novel engineered meganuclease induces homologous recombination in yeast and mammalian cells

21 January 2018

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Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

21 January 2018

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Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

21 January 2018

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A TALE nuclease architecture for efficient genome editing

21 January 2018

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Endonucleases: tools to correct the dystrophin gene.

21 January 2018

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Meganucleases can restore the reading frame of a mutated dystrophin

21 January 2018

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Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals

21 January 2018

1 reference

Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors

21 January 2018

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Very mild muscular dystrophy associated with the deletion of 46% of dystrophin

21 January 2018

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rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice.

21 January 2018

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Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury.

21 January 2018

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Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy

21 January 2018

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Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

21 January 2018

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Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice

21 January 2018

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Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

21 January 2018

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Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart

21 January 2018

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Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart.

21 January 2018

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Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies

21 January 2018

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Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog.

21 January 2018

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A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs.

21 January 2018

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Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery

21 January 2018

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Transduction efficiency and immune response associated with the administration of AAV8 vector into dog skeletal muscle

21 January 2018

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Immune responses to AAV in canine muscle monitored by cellular assays and noninvasive imaging

21 January 2018

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Injection of a recombinant AAV serotype 2 into canine skeletal muscles evokes strong immune responses against transgene products

21 January 2018

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AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

21 January 2018

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Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

21 January 2018

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Dystrophin immunity in Duchenne's muscular dystrophy.

21 January 2018

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Systemic Trans-splicing adeno-associated viral delivery efficiently transduces the heart of adult mdx mouse, a model for duchenne muscular dystrophy

21 January 2018

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Gene therapy of mdx mice with large truncated dystrophins generated by recombination using rAAV6.

21 January 2018

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Novel Mini–Dystrophin Gene Dual Adeno-Associated Virus Vectors Restore Neuronal Nitric Oxide Synthase Expression at the Sarcolemma

21 January 2018

1 reference

The cell biology of disease: FSHD: copy number variations on the theme of muscular dystrophy

21 January 2018

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AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy

21 January 2018

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A naturally occurring human minidysferlin protein repairs sarcolemmal lesions in a mouse model of dysferlinopathy.

21 January 2018

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Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer

21 January 2018

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Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.

21 January 2018

1 reference

Viral gene delivery to skeletal muscle: insights on maturation-dependent loss of fiber infectivity for adenovirus and herpes simplex type 1 viral vectors

21 January 2018

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Herpes simplex virus type 1 amplicon vector-mediated gene transfer to muscle

21 January 2018

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Herpes simplex virus vector-mediated dystrophin gene transfer and expression in MDX mouse skeletal muscle

21 January 2018

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Progress and prospects: immune responses to viral vectors

21 January 2018

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Capsid-modified adenoviral vectors for improved muscle-directed gene therapy

21 January 2018

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Correction of the dystrophic phenotype by in vivo targeting of muscle progenitor cells

21 January 2018

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Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs

21 January 2018

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Expression of dog microdystrophin in mouse and dog muscles by gene therapy

21 January 2018

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Autologous transplantation of muscle precursor cells modified with a lentivirus for muscular dystrophy: human cells and primate models

21 January 2018

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Muscle engraftment of myogenic progenitor cells following intraarterial transplantation

21 January 2018

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Autologous transplantation of SM/C-2.6( ) satellite cells transduced with micro-dystrophin CS1 cDNA by lentiviral vector into mdx mice.

21 January 2018

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Dystrophin delivery to muscles of mdx mice using lentiviral vectors leads to myogenic progenitor targeting and stable gene expression.

21 January 2018

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Transplantation of genetically corrected human iPSC-derived progenitors in mice with limb-girdle muscular dystrophy.

21 January 2018

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Retroviral integrations in gene therapy trials

21 January 2018

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Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy.

21 January 2018

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Dose Response in Rodents and Nonhuman Primates After Hydrodynamic Limb Vein Delivery of Naked Plasmid DNA

21 January 2018

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Functional efficacy of dystrophin expression from plasmids delivered to mdx mice by hydrodynamic limb vein injection

21 January 2018

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Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophy.

21 January 2018

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Gene transfer into muscle by electroporation in vivo.

21 January 2018

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High-efficiency gene transfer into skeletal muscle mediated by electric pulses

21 January 2018

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Electrotransfer of the full-length dog dystrophin into mouse and dystrophic dog muscles

21 January 2018

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Human Artificial Chromosomes for Gene Delivery and the Development of Animal Models

21 January 2018

1 reference

A highly stable and nonintegrated human artificial chromosome (HAC) containing the 2.4 Mb entire human dystrophin gene

21 January 2018

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Complete genetic correction of ips cells from Duchenne muscular dystrophy

21 January 2018

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Stem cell-mediated transfer of a human artificial chromosome ameliorates muscular dystrophy.

21 January 2018

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Recent developments in transposon-mediated gene therapy

21 January 2018

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Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

21 January 2018

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Restoration of dystrophin expression using the Sleeping Beauty transposon.

21 January 2018

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Novel hyperactive transposons for genetic modification of induced pluripotent and adult stem cells: a nonviral paradigm for coaxed differentiation.

21 January 2018

1 reference

Intrinsic cell memory reinforces myogenic commitment of pericyte-derived iPSCs

21 January 2018

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Repairing skeletal muscle: regenerative potential of skeletal muscle stem cells

21 January 2018

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Stem cell therapies for muscle disorders

21 January 2018

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Satellite cell of skeletal muscle fibers

21 January 2018

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Asymmetric self-renewal and commitment of satellite stem cells in muscle.

21 January 2018

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Wnt7a activates the planar cell polarity pathway to drive the symmetric expansion of satellite stem cells

21 January 2018

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Asymmetric division and cosegregation of template DNA strands in adult muscle satellite cells

21 January 2018

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A subpopulation of adult skeletal muscle stem cells retains all template DNA strands after cell division

21 January 2018

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Muscle satellite cells adopt divergent fates: a mechanism for self-renewal?

21 January 2018

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Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts

21 January 2018

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Intramuscular cell transplantation as a potential treatment of myopathies: clinical and preclinical relevant data

21 January 2018

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Tissue-specific stem cells: lessons from the skeletal muscle satellite cell

21 January 2018

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Defective myoblasts identified in Duchenne muscular dystrophy

21 January 2018

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Disruption of DAG1 in differentiated skeletal muscle reveals a role for dystroglycan in muscle regeneration.

21 January 2018

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Satellite cell senescence underlies myopathy in a mouse model of limb-girdle muscular dystrophy 2H.

21 January 2018

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Short telomeres and stem cell exhaustion model Duchenne muscular dystrophy in mdx/mTR mice

21 January 2018

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Alpha sarcoglycan is required for FGF-dependent myogenic progenitor cell proliferation in vitro and in vivo

21 January 2018

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Premature proliferative arrest of cricopharyngeal myoblasts in oculo-pharyngeal muscular dystrophy: Therapeutic perspectives of autologous myoblast transplantation

21 January 2018

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Myogenic specification of side population cells in skeletal muscle

21 January 2018

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Identification of a novel population of muscle stem cells in mice: potential for muscle regeneration

21 January 2018

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Hematopoietic potential of stem cells isolated from murine skeletal muscle

21 January 2018

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Contribution of Human Muscle-Derived Cells to Skeletal Muscle Regeneration in Dystrophic Host Mice

21 January 2018

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Systemic delivery of allogenic muscle stem cells induces long-term muscle repair and clinical efficacy in duchenne muscular dystrophy dogs

21 January 2018

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Human circulating AC133( ) stem cells restore dystrophin expression and ameliorate function in dystrophic skeletal muscle

21 January 2018

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Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice

21 January 2018

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Cell Therapy of -Sarcoglycan Null Dystrophic Mice Through Intra-Arterial Delivery of Mesoangioblasts

21 January 2018

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PlGF-MMP-9-expressing cells restore microcirculation and efficacy of cell therapy in aged dystrophic muscle

21 January 2018

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Partial dysferlin reconstitution by adult murine mesoangioblasts is sufficient for full functional recovery in a murine model of dysferlinopathy.

21 January 2018

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Complete repair of dystrophic skeletal muscle by mesoangioblasts with enhanced migration ability

21 January 2018

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Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells

21 January 2018

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Pericytes resident in postnatal skeletal muscle differentiate into muscle fibres and generate satellite cells

21 January 2018

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The increase of pericyte population in human neuromuscular disorders supports their role in muscle regeneration in vivo

21 January 2018

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Satellite cells are essential for skeletal muscle regeneration: the cell on the edge returns centre stage

21 January 2018

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Mesoangioblasts suppress T cell proliferation through IDO and PGE-2-dependent pathways

21 January 2018

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Quantitative muscle strength assessment in duchenne muscular dystrophy: longitudinal study and correlation with functional measures

21 January 2018

1 reference

'Hearts and bones': the ups and downs of 'plasticity' in stem cell biology.

21 January 2018

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Bone marrow stromal cells generate muscle cells and repair muscle degeneration.

21 January 2018

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A perivascular origin for mesenchymal stem cells in multiple human organs

21 January 2018

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Engraftment of mesenchymal stem cells into dystrophin-deficient mice is not accompanied by functional recovery

21 January 2018

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The vast majority of bone-marrow-derived cells integrated into mdx muscle fibers are silent despite long-term engraftment

21 January 2018

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Mesenchymal stem cells as anti-inflammatories: implications for treatment of Duchenne muscular dystrophy.

21 January 2018

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Skeletal muscle repair by adult human mesenchymal stem cells from synovial membrane.

21 January 2018

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The contribution of human synovial stem cells to skeletal muscle regeneration

21 January 2018

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The meaning, the sense and the significance: translating the science of mesenchymal stem cells into medicine

21 January 2018

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Muscle regeneration by bone marrow-derived myogenic progenitors

21 January 2018

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Failure to correct murine muscular dystrophy

21 January 2018

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Long-term persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation

21 January 2018

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Muscle-derived hematopoietic stem cells are hematopoietic in origin

21 January 2018

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Establishment in culture of pluripotential cells from mouse embryos

21 January 2018

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Embryonic stem cell lines derived from human blastocysts

21 January 2018

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Derivation of engraftable skeletal myoblasts from human embryonic stem cells

21 January 2018

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Functional skeletal muscle regeneration from differentiating embryonic stem cells

21 January 2018

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Assessment of the myogenic stem cell compartment following transplantation of Pax3/Pax7-induced embryonic stem cell-derived progenitors

21 January 2018

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Induced pluripotent stem cells: past, present, and future

21 January 2018

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Harnessing the potential of induced pluripotent stem cells for regenerative medicine

21 January 2018

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Human ES- and iPS-derived myogenic progenitors restore DYSTROPHIN and improve contractility upon transplantation in dystrophic mice.

21 January 2018

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Myoblasts derived from normal hESCs and dystrophic hiPSCs efficiently fuse with existing muscle fibers following transplantation

21 January 2018

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Virally mediated gene manipulation in the adult CNS.

21 January 2018

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Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins

21 January 2018

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Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs

21 January 2018

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Dystrophin expression in the mdx mouse restored by stem cell transplantation

21 January 2018

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21 January 2018

Identifiers

DOI

10.1111/FEBS.12178

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28 August 2017

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