Progress and prospects of gene therapy clinical trials for the muscular dystrophies. (Q34497471)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

title

Progress and prospects of gene therapy clinical trials for the muscular dystrophies (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

0 references

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

Jeffrey S Chamberlain

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

author name string

Niclas E Bengtsson

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

John K Hall

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

Guy L Odom

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

publication date

8 October 2015

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

published in

Human Molecular Genetics

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

volume

25

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

issue

R1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

page(s)

R9-17

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

Human α7 Integrin Gene (ITGA7) Delivered by Adeno-Associated Virus Extends Survival of Severely Affected Dystrophin/Utrophin-Deficient Mice

26 February 2020

cites work

1 reference

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AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models

7 July 2018

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Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.

7 July 2018

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Intra-amniotic rAAV-mediated microdystrophin gene transfer improves canine X-linked muscular dystrophy and may induce immune tolerance

7 July 2018

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Engraftment potential of dermal fibroblasts following in vivo myogenic conversion in immunocompetent dystrophic skeletal muscle

7 July 2018

1 reference

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Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9.

7 July 2018

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Regional delivery of mesothelin-targeted CAR T cell therapy generates potent and long-lasting CD4-dependent tumor immunity

7 July 2018

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A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.

7 July 2018

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Characterization of dystrophin deficient rats: a new model for Duchenne muscular dystrophy

7 July 2018

1 reference

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CRISPR-Cas9 knockin mice for genome editing and cancer modeling

7 July 2018

1 reference

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Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.

7 July 2018

1 reference

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Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

7 July 2018

1 reference

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Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice

7 July 2018

1 reference

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Muscle and heart function restoration in a limb girdle muscular dystrophy 2I (LGMD2I) mouse model by systemic FKRP gene delivery

7 July 2018

1 reference

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Generation of muscular dystrophy model rats with a CRISPR/Cas system

7 July 2018

1 reference

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Myocyte-mediated arginase expression controls hyperargininemia but not hyperammonemia in arginase-deficient mice

7 July 2018

1 reference

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Removing T-cell epitopes with computational protein design.

7 July 2018

1 reference

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Tolerance induction in memory CD4 T cells requires two rounds of antigen-specific activation

7 July 2018

1 reference

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Recombinant immunotoxin for cancer treatment with low immunogenicity by identification and silencing of human T-cell epitopes

7 July 2018

1 reference

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Adoptive therapy with chimeric antigen receptor-modified T cells of defined subset composition

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Efficacy and toxicity management of 19-28z CAR T cell therapy in B cell acute lymphoblastic leukemia

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Elimination of contaminating cap genes in AAV vector virions reduces immune responses and improves transgene expression in a canine gene therapy model

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Design and implementation of adoptive therapy with chimeric antigen receptor-modified T cells

7 July 2018

1 reference

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Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Genome engineering using the CRISPR-Cas9 system

7 July 2018

1 reference

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Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogates.

7 July 2018

1 reference

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Donor-derived CD19-targeted T cells cause regression of malignancy persisting after allogeneic hematopoietic stem cell transplantation

7 July 2018

1 reference

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Anti-dystrophin T cell responses in Duchenne muscular dystrophy: prevalence and a glucocorticoid treatment effect

7 July 2018

1 reference

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Adeno-associated virus 9 mediated FKRP gene therapy restores functional glycosylation of α-dystroglycan and improves muscle functions

7 July 2018

1 reference

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Plasmid-encoded proinsulin preserves C-peptide while specifically reducing proinsulin-specific CD8⁺ T cells in type 1 diabetes

7 July 2018

1 reference

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Immune responses to AAV vectors: overcoming barriers to successful gene therapy

7 July 2018

1 reference

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Chimeric antigen receptor-modified T cells for acute lymphoid leukemia

7 July 2018

1 reference

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Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy.

7 July 2018

1 reference

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CD19-targeted T cells rapidly induce molecular remissions in adults with chemotherapy-refractory acute lymphoblastic leukemia

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Identification of infants at risk for developing Fabry, Pompe, or mucopolysaccharidosis-I from newborn blood spots by tandem mass spectrometry

7 July 2018

1 reference

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A comprehensive review of retinal gene therapy

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

AAV-mediated overexpression of human α7 integrin leads to histological and functional improvement in dystrophic mice

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Microdystrophin ameliorates muscular dystrophy in the canine model of duchenne muscular dystrophy

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Follistatin-mediated skeletal muscle hypertrophy is regulated by Smad3 and mTOR independently of myostatin

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Characteristics of rare disease marketing applications associated with FDA product approvals 2006-2010.

7 July 2018

1 reference

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MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Next generation sequencing for molecular diagnosis of neuromuscular diseases.

7 July 2018

1 reference

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Translating the therapeutic potential of neurotrophic factors to clinical 'proof of concept': a personal saga achieving a career-long quest

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Transcription factor rational design improves directed differentiation of human mesenchymal stem cells into skeletal myocytes.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Stem-cell gene therapy for the Wiskott-Aldrich syndrome

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Partial rescue of growth failure in growth hormone (GH)-deficient mice by a single injection of a double-stranded adeno-associated viral vector expressing the GH gene driven by a muscle-specific regulatory cassette

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector-mediated gene therapy

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Clinical trials with oncolytic adenovirus in China

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Obstructive nephropathy: towards biomarker discovery and gene therapy

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Transgenic expression of {alpha}7{beta}1 integrin maintains muscle integrity, increases regenerative capacity, promotes hypertrophy, and reduces cardiomyopathy in dystrophic mice

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Cancer fears cast doubts on future of gene therapy

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

The danger model: a renewed sense of self

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Successful readministration of adeno-associated virus vectors to the mouse lung requires transient immunosuppression during the initial exposure

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Gene therapy for human genetic disease?

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4802376

Gene therapy deserves a fresh chance

7 July 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV420

Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV420

Vascular endothelial growth factor as a biomarker for endostatin gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV420

Biomarkers Predict p53 Gene Therapy Efficacy in Recurrent Squamous Cell Carcinoma of the Head and Neck

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV420

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV420

AAV-mediated gene therapy to the isolated limb in rhesus macaques

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV420

Design and testing of regulatory cassettes for optimal activity in skeletal and cardiac muscles

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV420

Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV420

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV420

Dystrophin immunity in Duchenne's muscular dystrophy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV420

Cancer fears cast doubts on future of gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDV420

Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26450518

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26450518

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Heparan sulfate and dermatan sulfate derived disaccharides are sensitive markers for newborn screening for mucopolysaccharidoses types I, II and III

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26450518

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1093/HMG/DDV420

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json

26 February 2020

PubMed publication ID

26450518

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26450518%20AND%20SRC:MED&resulttype=core&format=json