Successful regional delivery and long-term expression of a dystrophin gene in canine muscular dystrophy: a preclinical model for human therapies (Q36142496)

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

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scholarly article

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Europe PubMed Central

PMC publication ID

12 August 2017

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

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Europe PubMed Central

PMC publication ID

12 August 2017

muscular dystrophy

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inferred from title

2

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Rainer Storb

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Europe PubMed Central

12 August 2017

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A. Dusty Miller

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A Dusty Miller

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12 August 2017

Christine L Halbert

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Christine L Halbert

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12 August 2017

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1

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Zejing Wang

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Europe PubMed Central

12 August 2017

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author name string

Glen B Banks

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12 August 2017

Tiffany M Butts

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12 August 2017

Eric E Finn

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12 August 2017

James M Allen

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12 August 2017

Jeffrey S Chamberlain

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12 August 2017

Stephen J Tapscott

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12 August 2017

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12 June 2012

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12 August 2017

Molecular Therapy

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12 August 2017

20

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12 August 2017

8

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12 August 2017

1501-1507

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Europe PubMed Central

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12 August 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Immune Responses to rAAV6: The Influence of Canine Parvovirus Vaccination and Neonatal Administration of Viral Vector

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Humoral immunity to AAV-6, 8, and 9 in normal and dystrophic dogs

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Cell therapy for muscular dystrophies: advances and challenges

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Neutralizing antibodies against AAV serotypes 1, 2, 6, and 9 in sera of commonly used animal models

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Restoring dystrophin expression in duchenne muscular dystrophy muscle progress in exon skipping and stop codon read through

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Current advances in cell therapy strategies for muscular dystrophies

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

New insights in gene-derived therapy: the example of Duchenne muscular dystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Past, present and future of myoblast transplantation in the treatment of Duchenne muscular dystrophy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Expression of human alpha1-antitrypsin in mice and dogs following AAV6 vector-mediated gene transfer to the lungs.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Evaluation of vascular delivery methodologies to enhance rAAV6-mediated gene transfer to canine striated musculature

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Gene therapy in large animal models of muscular dystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Molecular and cellular adaptations to chronic myotendinous strain injury in mdx mice expressing a truncated dystrophin

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: Implications for gene therapy using AAV vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

The dystrophin glycoprotein complex: signaling strength and integrity for the sarcolemma

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Forced expression of dystrophin deletion constructs reveals structure-function correlations.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

The structural and functional diversity of dystrophin

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Membrane organization of the dystrophin-glycoprotein complex

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Force transmission across muscle cell membranes

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 October 2017

Treatment of established graft-versus-host disease in dogs by antithymocyte serum or prednisone

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 July 2018

Treatment of established human graft-versus-host disease by antithymocyte globulin.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 July 2018

Dystrophin is required for normal thin filament-membrane associations at myotendinous junctions

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

2 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

23 September 2018

Dystrophin immunity in Duchenne's muscular dystrophy.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

23 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

23 September 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

23 September 2018

CD8(+) T-cell responses to adeno-associated virus capsid in humans

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

23 September 2018

Immunity to adeno-associated virus-mediated gene transfer in a random-bred canine model of Duchenne muscular dystrophy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

23 September 2018

Cyclophosphamide and antithymocyte globulin as a conditioning regimen for allogeneic marrow transplantation in patients with aplastic anaemia: a long-term follow-up

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

23 September 2018

Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3412492

23 September 2018

Effect of prior blood transfusions on marrow grafts: abrogation of sensitization by procarbazine and antithymocyte serum

1 reference

https://pubmed.ncbi.nlm.nih.gov/22692496

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Hematopoietic stem cell transplantation does not restore dystrophin expression in Duchenne muscular dystrophy dogs

1 reference

https://pubmed.ncbi.nlm.nih.gov/22692496

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Stable mixed hematopoietic chimerism in DLA-identical littermate dogs given sublethal total body irradiation before and pharmacological immunosuppression after marrow transplantation

1 reference

https://pubmed.ncbi.nlm.nih.gov/22692496

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Cyclophosphamide combined with antithymocyte globulin in preparation for allogeneic marrow transplants in patients with aplastic anemia

1 reference

https://pubmed.ncbi.nlm.nih.gov/22692496

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/22692496

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Synergism between mycophenolate mofetil and cyclosporine in preventing graft-versus-host disease among lethally irradiated dogs given DLA-nonidentical unrelated marrow grafts

1 reference

https://pubmed.ncbi.nlm.nih.gov/22692496

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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10.1038/MT.2012.111

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Europe PubMed Central

PMC publication ID

12 August 2017

PMC publication ID

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Europe PubMed Central

PMC publication ID

12 August 2017

PubMed publication ID

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Europe PubMed Central

PMC publication ID

12 August 2017

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