Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
Bengtsson, Niclas E., Hall, John K., Odom, Guy L., Phelps, Michael P., Andrus, Colin R., Hawkins, R. David, Hauschka, Stephen D., Chamberlain, Joel R., Chamberlain, Jeffrey S.
Published in Nature communications (14.02.2017)
Published in Nature communications (14.02.2017)
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Journal Article
Biomarkers for Duchenne muscular dystrophy progression: impact of age in the mdx tongue spared muscle
Lorena, Marcelo dos Santos Voltani, Santos, Estela Kato dos, Ferretti, Renato, Nagana Gowda, G. A, Odom, Guy L, Chamberlain, Jeffrey S, Matsumura, Cintia Yuri
Published in Skeletal muscle (13.09.2023)
Published in Skeletal muscle (13.09.2023)
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Journal Article
Recombinant adeno‐associated viral (rAAV) vectors mediate efficient gene transduction in cultured neonatal and adult microglia
Su, Wei, Kang, John, Sopher, Bryce, Gillespie, James, Aloi, Macarena S., Odom, Guy L., Hopkins, Stephanie, Case, Amanda, Wang, David B., Chamberlain, Jeffrey S., Garden, Gwenn A.
Published in Journal of neurochemistry (01.01.2016)
Published in Journal of neurochemistry (01.01.2016)
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Journal Article
Muscle structure influences utrophin expression in mdx mice
Banks, Glen B, Combs, Ariana C, Odom, Guy L, Bloch, Robert J, Chamberlain, Jeffrey S
Published in PLoS genetics (01.06.2014)
Published in PLoS genetics (01.06.2014)
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Journal Article
Dystrobrevin increases dystrophin's binding to the dystrophin–glycoprotein complex and provides protection during cardiac stress
Strakova, Jana, Dean, Jon D, Sharpe, Katharine M, Meyers, Tatyana A, Odom, Guy L, Townsend, DeWayne
Published in Journal of molecular and cellular cardiology (01.11.2014)
Published in Journal of molecular and cellular cardiology (01.11.2014)
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Journal Article
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model
Piepho, Arden B., Lowe, Jeovanna, Cumby, Laurel R., Dorn, Lisa E., Lake, Dana M., Rastogi, Neha, Gertzen, Megan D., Sturgill, Sarah L., Odom, Guy L., Ziolo, Mark T., Accornero, Federica, Chamberlain, Jeffrey S., Rafael-Fortney, Jill A.
Published in Molecular therapy. Methods & clinical development (09.03.2023)
Published in Molecular therapy. Methods & clinical development (09.03.2023)
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Journal Article
Micro-dystrophin gene therapy prevents heart failure in an improved Duchenne muscular dystrophy cardiomyopathy mouse model
Howard, Zachary M, Dorn, Lisa E, Lowe, Jeovanna, Gertzen, Megan D, Ciccone, Pierce, Rastogi, Neha, Odom, Guy L, Accornero, Federica, Chamberlain, Jeffrey S, Rafael-Fortney, Jill A
Published in JCI insight (08.04.2021)
Published in JCI insight (08.04.2021)
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Journal Article
Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors
Seto, Jane T, Ramos, Julian N, Muir, Lindsey, Chamberlain, Jeffrey S, Odom, Guy L
Published in Current gene therapy (01.06.2012)
Published in Current gene therapy (01.06.2012)
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Journal Article
Gene Therapy Rescues Cardiac Dysfunction in Duchenne Muscular Dystrophy Mice by Elevating Cardiomyocyte Deoxy-Adenosine Triphosphate
Kolwicz, Stephen C., Hall, John K., Moussavi-Harami, Farid, Chen, Xiolan, Hauschka, Stephen D., Chamberlain, Jeffrey S., Regnier, Michael, Odom, Guy L.
Published in JACC. Basic to translational science (01.11.2019)
Published in JACC. Basic to translational science (01.11.2019)
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Journal Article
Translation of Cardiac Myosin Activation With 2-Deoxy-ATP to Treat Heart Failure Via an Experimental Ribonucleotide Reductase-Based Gene Therapy
Thomson, Kassandra S., PhD, Odom, Guy L., PhD, Murry, Charles E., MD, PhD, Mahairas, Gregory G., PhD, Moussavi-Harami, Farid, MD, Teichman, Sam L., MD, Chen, Xiaolan, PhD, Hauschka, Stephen D., PhD, Chamberlain, Jeffrey S., PhD, Regnier, Michael, PhD
Published in JACC. Basic to translational science (01.12.2016)
Published in JACC. Basic to translational science (01.12.2016)
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Journal Article
Progress and prospects of gene therapy clinical trials for the muscular dystrophies
Bengtsson, Niclas E, Seto, Jane T, Hall, John K, Chamberlain, Jeffrey S, Odom, Guy L
Published in Human molecular genetics (15.04.2016)
Published in Human molecular genetics (15.04.2016)
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Journal Article
Gene Therapy of mdx Mice With Large Truncated Dystrophins Generated by Recombination Using rAAV6
Odom, Guy L, Gregorevic, Paul, Allen, James M, Chamberlain, Jeffrey S
Published in Molecular therapy (01.01.2011)
Published in Molecular therapy (01.01.2011)
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Journal Article
AAV6-mediated Cardiac-specific Overexpression of Ribonucleotide Reductase Enhances Myocardial Contractility
Kolwicz, Stephen C, Odom, Guy L, Nowakowski, Sarah G, Moussavi-Harami, Farid, Chen, Xiaolan, Reinecke, Hans, Hauschka, Stephen D, Murry, Charles E, Mahairas, Gregory G, Regnier, Michael
Published in Molecular therapy (01.02.2016)
Published in Molecular therapy (01.02.2016)
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Journal Article
Corrigendum: Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
Bengtsson, Niclas E, Hall, John K, Odom, Guy L, Phelps, Michael P, Andrus, Colin R, Hawkins, R David, Hauschka, Stephen D, Chamberlain, Joel R, Chamberlain, Jeffrey S
Published in Nature communications (23.06.2017)
Published in Nature communications (23.06.2017)
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Journal Article
Correction: Corrigendum: Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
Bengtsson, Niclas E., Hall, John K., Odom, Guy L., Phelps, Michael P., Andrus, Colin R., Hawkins, R. David, Hauschka, Stephen D., Chamberlain, Joel R., Chamberlain, Jeffrey S.
Published in Nature communications (23.06.2017)
Published in Nature communications (23.06.2017)
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