(Q36506919)

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Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy

scientific article published on 27 January 2016

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scholarly article

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy (English)

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

Hanns Lochmüller

7

object named as

Hanns Lochmüller

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

author name string

Jinhong Meng

1

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

John R Counsell

2

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

Mojgan Reza

3

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

Steven H Laval

4

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

Olivier Danos

5

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

Adrian Thrasher

6

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

Francesco Muntoni

8

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

Jennifer E Morgan

9

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Europe PubMed Central

PMC publication ID

14 August 2017

language of work or name

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publication date

27 January 2016

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Europe PubMed Central

PMC publication ID

14 August 2017

Scientific Reports

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Europe PubMed Central

PMC publication ID

14 August 2017

6

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

19750

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

copyright license

Creative Commons Attribution 4.0 International

27 January 2016

1 reference

April 2022 Public Data File from Crossref

copyright status

0 references

Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD).

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

How much dystrophin is enough: the physiological consequences of different levels of dystrophin in the mdx mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Adeno-associated viral (AAV) vectors do not efficiently target muscle satellite cells

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Human skeletal muscle-derived CD133(+) cells form functional satellite cells after intramuscular transplantation in immunodeficient host mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo

1 reference

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30 September 2017

Revertant fibers in the mdx murine model of Duchenne muscular dystrophy: an age- and muscle-related reappraisal

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Identification of new dystroglycan complexes in skeletal muscle

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Green fluorescent protein expression triggers proteome changes in breast cancer cells.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

A new immuno-, dystrophin-deficient model, the NSG-mdx(4Cv) mouse, provides evidence for functional improvement following allogeneic satellite cell transplantation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

α2 and α3 helices of dystrophin R16 and R17 frame a microdomain in the α1 helix of dystrophin R17 for neuronal NOS binding

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Understanding lentiviral vector chromatin targeting: working to reduce insertional mutagenic potential for gene therapy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

The effects of low levels of dystrophin on mouse muscle function and pathology

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Marginal level dystrophin expression improves clinical outcome in a strain of dystrophin/utrophin double knockout mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Physiological characterization of muscle strength with variable levels of dystrophin restoration in mdx mice following local antisense therapy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

The contribution of human synovial stem cells to skeletal muscle regeneration

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Desmin-regulated lentiviral vectors for skeletal muscle gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

In vivo myogenic potential of human CD133+ muscle-derived stem cells: a quantitative study

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Sub-physiological sarcoglycan expression contributes to compensatory muscle protection in mdx mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Highly efficient, functional engraftment of skeletal muscle stem cells in dystrophic muscles

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Preservation of muscle force in Mdx3cv mice correlates with low-level expression of a near full-length dystrophin protein

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Stable gene transfer to muscle using non-integrating lentiviral vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

The dark side of EGFP: defective polyubiquitination

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Systematic determination of the packaging limit of lentiviral vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Assembly of the dystrophin-associated protein complex does not require the dystrophin COOH-terminal domain

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

A third-generation lentivirus vector with a conditional packaging system

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Nitric oxide synthase complexed with dystrophin and absent from skeletal muscle sarcolemma in Duchenne muscular dystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

30 September 2017

The human desmin promoter drives robust gene expression for skeletal muscle stem cell-mediated gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

28 June 2018

Somatic reversion/suppression of the mouse mdx phenotype in vivo.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

28 June 2018

Dystrophin-associated proteins are greatly reduced in skeletal muscle from mdx mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

28 June 2018

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

21 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

21 September 2018

Optimized production and concentration of lentiviral vectors containing large inserts.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

21 September 2018

Dystrophinopathy carrier determination and detection of protein deficiencies in muscular dystrophy using lentiviral MyoD-forced myogenesis.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

21 September 2018

Absence of neuronal nitric oxide synthase (nNOS) as a pathological marker for the diagnosis of Becker muscular dystrophy with rod domain deletions

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

21 September 2018

ZZ domain is essentially required for the physiological binding of dystrophin and utrophin to beta-dystroglycan

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

21 September 2018

Relocalization of neuronal nitric oxide synthase (nNOS) as a marker for complete restoration of the dystrophin associated protein complex in skeletal muscle

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

21 September 2018

Mini- and full-length dystrophin gene transfer induces the recovery of nitric oxide synthase at the sarcolemma of mdx4cv skeletal muscle fibers

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4728433

21 September 2018

CD47(low) status on CD4 effectors is necessary for the contraction/resolution of the immune response in humans and mice.

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21 January 2018

Human Muscle Precursor Cell Regeneration in the Mouse Host Is Enhanced by Growth Factors

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https://api.crossref.org/works/10.1038%2FSREP19750

21 January 2018

Evidence-based path to newborn screening for Duchenne muscular dystrophy

1 reference

https://pubmed.ncbi.nlm.nih.gov/26813695

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Revertant fibres: a possible genetic therapy for Duchenne muscular dystrophy?

1 reference

https://pubmed.ncbi.nlm.nih.gov/26813695

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne's or Becker's muscular dystrophy

1 reference

https://pubmed.ncbi.nlm.nih.gov/26813695

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Human muscle precursor cells give rise to functional satellite cells in vivo

1 reference

https://pubmed.ncbi.nlm.nih.gov/26813695

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1038/SREP19750

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

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0 references

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

PubMed publication ID

1 reference

Europe PubMed Central

PMC publication ID

14 August 2017

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