(Q38079633)

28 August 2017

title

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

1 reference

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

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

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

21 January 2018

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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

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

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

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

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

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

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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https://api.crossref.org/works/10.1111%2FFEBS.12178

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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https://api.crossref.org/works/10.1111%2FFEBS.12178

PTC124 targets genetic disorders caused by nonsense mutations

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

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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https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

Homing endonuclease structure and function

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

A TALE nuclease architecture for efficient genome editing

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

Endonucleases: tools to correct the dystrophin gene.

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

Meganucleases can restore the reading frame of a mutated dystrophin

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

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

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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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https://api.crossref.org/works/10.1111%2FFEBS.12178

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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https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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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https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Dystrophin immunity in Duchenne's muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Novel mini-dystrophin gene dual adeno-associated virus vectors restore neuronal nitric oxide synthase expression at the sarcolemma

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Progress and prospects: immune responses to viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Muscle engraftment of myogenic progenitor cells following intraarterial transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Retroviral integrations in gene therapy trials

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Dose response in rodents and nonhuman primates after hydrodynamic limb vein delivery of naked plasmid DNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

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https://api.crossref.org/works/10.1111%2FFEBS.12178

Gene transfer into muscle by electroporation in vivo.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Human artificial chromosomes for gene delivery and the development of animal models

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Complete genetic correction of ips cells from Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Recent developments in transposon-mediated gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Restoration of dystrophin expression using the Sleeping Beauty transposon.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Intrinsic cell memory reinforces myogenic commitment of pericyte-derived iPSCs

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Repairing skeletal muscle: regenerative potential of skeletal muscle stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Stem cell therapies for muscle disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Satellite cell of skeletal muscle fibers

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Defective myoblasts identified in Duchenne muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Myogenic specification of side population cells in skeletal muscle

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Hematopoietic potential of stem cells isolated from murine skeletal muscle

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Contribution of human muscle-derived cells to skeletal muscle regeneration in dystrophic host mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

A perivascular origin for mesenchymal stem cells in multiple human organs

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

The contribution of human synovial stem cells to skeletal muscle regeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Muscle regeneration by bone marrow-derived myogenic progenitors

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Failure to correct murine muscular dystrophy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Muscle-derived hematopoietic stem cells are hematopoietic in origin

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Establishment in culture of pluripotential cells from mouse embryos

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Embryonic stem cell lines derived from human blastocysts

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Derivation of engraftable skeletal myoblasts from human embryonic stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Functional skeletal muscle regeneration from differentiating embryonic stem cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Induced pluripotent stem cells: past, present, and future

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Harnessing the potential of induced pluripotent stem cells for regenerative medicine

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Virally mediated gene manipulation in the adult CNS.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

Dystrophin expression in the mdx mouse restored by stem cell transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FFEBS.12178

21 January 2018

Identifiers

DOI

10.1111/FEBS.12178

1 reference

28 August 2017

1 reference