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List of works by Jeffrey S Chamberlain

242 Breakpoints in the 200-kb deletion-prone P20 region of the DMD gene are widely spread ⬇️

scientific article published on July 1, 1991

A Genetic Intervention Stands a Skip Away from Clinical Tests ⬇️

scientific article published on December 14, 2012

A highly functional mini-dystrophin/GFP fusion gene for cell and gene therapy studies of Duchenne muscular dystrophy

scientific article published on 4 April 2006

Age-dependent dystrophin loss and genetic reconstitution establish a molecular link between dystrophin and heart performance during aging.

scientific article

An Epitope Structure for the C-Terminal Domain of Dystrophin and Utrophin ⬇️

scientific article published on August 4, 1998

An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells ⬇️

scientific article published on January 1, 2013

Cell-lineage regulated myogenesis for dystrophin replacement: a novel therapeutic approach for treatment of muscular dystrophy

scientific article published on 29 May 2008

Contractile properties of diaphragm muscle segments from old mdx and old transgenic mdx mice ⬇️

scientific article published on 01 June 1997

Development of Novel Micro-dystrophins with Enhanced Functionality

scientific article published on 01 February 2019

Duchenne Muscular Dystrophy Models Show Their Age ⬇️

scientific article published on December 23, 2010

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

scientific article

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

scientific article published on 23 February 2009

Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy

article published in the Proceedings of the National Academy of Sciences of the United States of America

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

scientific article

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

scientific article published on August 1, 1995

Expression of recombinant dystrophin and its localization to the cell membrane ⬇️

scientific article published on January 24, 1991

Expression of the dystrophin isoform Dp116 preserves functional muscle mass and extends lifespan without preventing dystrophy in severely dystrophic mice

scientific article published on 23 September 2011

Extracorporeal delivery of rAAV with metabolic exchange and oxygenation

scientific article published on January 2013

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

scientific article published on June 18, 2012

Functional deficits in nNOSmu-deficient skeletal muscle: myopathy in nNOS knockout mice

scientific article

Gene Replacement Therapies for Duchenne Muscular Dystrophy Using Adeno-Associated Viral Vectors ⬇️

scientific article published on June 1, 2012

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

scientific article

Heparin-binding correlates with increased efficiency of AAV1- and AAV6-mediated transduction of striated muscle, but negatively impacts CNS transduction ⬇️

scientific article published on 02 August 2012

Immune Evasion by Muscle-Specific Gene Expression in Dystrophic Muscle ⬇️

scientific article published on December 1, 2001

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

scientific article published on 3 November 2011

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

scientific article published on September 26, 2011

Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy ⬇️

scientific article published on July 1, 1997

Increased sphingosine-1-phosphate improves muscle regeneration in acutely injured mdx mice ⬇️

scientific article published on August 1, 2013

Is the carboxyl-terminus of dystrophin required for membrane association? A novel, severe case of duchenne muscular dystrophy ⬇️

scientific article published on October 1, 1991

Localization of Dlg at the mammalian neuromuscular junction ⬇️

scientific article published on June 22, 1998

Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/ Mice

scientific article published on 16 October 2018

Microutrophin expression in dystrophic mice displays myofiber type differences in therapeutic effects

scientific article published on 11 November 2020

Monitoring disease activity noninvasively in the model of Duchenne muscular dystrophy

article published in the Proceedings of the National Academy of Sciences of the United States of America

Muscle structure influences utrophin expression in mdx mice.

scientific article

Non-invasive tracking of disease progression in young dystrophic muscles using multi-parametric MRI at 14T

scientific article published in PLoS ONE

Onset of experimental severe cardiac fibrosis is mediated by overexpression of Angiotensin-converting enzyme 2.

scientific article published on 16 February 2009

Phosphorylation within the cysteine-rich region of dystrophin enhances its association with β-dystroglycan and identifies a potential novel therapeutic target for skeletal muscle wasting

scientific article

Production and Characterization of Improved Adenovirus Vectors with the E1, E2b, and E3 Genes Deleted ⬇️

scientific article published on February 1, 1998

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

scientific article

Prosurvival Factors Improve Functional Engraftment of Myogenically Converted Dermal Cells into Dystrophic Skeletal Muscle.

scientific article published on 8 August 2016

Stabilization of the cardiac sarcolemma by sarcospan rescues DMD-associated cardiomyopathy

scientific article published on 30 April 2019

Suppression of revertant fibers in mdx mice by expression of a functional dystrophin ⬇️

scientific article published on November 15, 2001

Surrogate gene therapy for muscular dystrophy

scientific article published on 01 October 2019

Sustained Human Factor VIII Expression in Hemophilia A Mice Following Systemic Delivery of a Gutless Adenoviral Vector ⬇️

scientific article published on January 1, 2002

Syntrophin binds directly to multiple spectrin-like repeats in dystrophin and mediates binding of nNOS to repeats 16-17

scientific article published on 01 September 2018

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

scientific article

Systemic microdystrophin gene delivery improves skeletal muscle structure and function in old dystrophic mdx mice.

scientific article published on 04 March 2008

Translation of Cardiac Myosin Activation with 2-deoxy-ATP to Treat Heart Failure via an Experimental Ribonucleotide Reductase-Based Gene Therapy

scientific article

Transplantation of myoblasts from a transgenic mouse overexpressing dystrophin produced only a relatively small increase of dystrophin-positive membrane ⬇️

scientific article published on January 1, 1998

Truncated dystrophins can influence neuromuscular synapse structure.

scientific article

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

scientific article

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