Search Results - "Chamberlain, Joel R"

Progress toward Gene Therapy for Duchenne Muscular Dystrophy by Chamberlain, Joel R., Chamberlain, Jeffrey S.

“…Duchenne muscular dystrophy (DMD) has been a major target for gene therapy development for nearly 30 years. DMD is among the most common genetic diseases, and…”
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Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy by 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.

“…Gene replacement therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne muscular dystrophy (DMD). A related approach…”
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Therapeutic impact of systemic AAV-mediated RNA interference in a mouse model of myotonic dystrophy by Bisset, Darren R, Stepniak-Konieczna, Ewa A, Zavaljevski, Maja, Wei, Jessica, Carter, Gregory T, Weiss, Michael D, Chamberlain, Joel R

“…RNA interference (RNAi) offers a promising therapeutic approach for dominant genetic disorders that involve gain-of-function mechanisms. One candidate disease…”
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Systemic RNAi delivery to the muscles of ROSA26 mice reduces lacZ expression by Wei, Jessica, Chamberlain, Joel R

“…RNAi has potential for therapeutically downregulating the expression of dominantly inherited genes in a variety of human genetic disorders. Here we used the…”
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Adeno-associated viral (AAV) vectors do not efficiently target muscle satellite cells by Arnett, Andrea Lh, Konieczny, Patryk, Ramos, Julian N, Hall, John, Odom, Guy, Yablonka-Reuveni, Zipora, Chamberlain, Joel R, Chamberlain, Jeffrey S

“…Adeno-associated viral (AAV) vectors are becoming an important tool for gene therapy of numerous genetic and other disorders. Several recombinant AAV vectors…”
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Molecular and cellular adaptations to chronic myotendinous strain injury in mdx mice expressing a truncated dystrophin by Banks, Glen B., Combs, Ariana C., Chamberlain, Joel R., Chamberlain, Jeffrey S.

“…Myotendinous strain injury is the most common injury of human skeletal muscles because the majority of muscle forces are transmitted through this region…”
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Corrigendum: Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy by 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

“…This corrects the article DOI: 10.1038/ncomms14454…”
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Endogenous MicroRNA Competition as a Mechanism of shRNA-Induced Cardiotoxicity by Course, Meredith M., Gudsnuk, Kathryn, Desai, Nitin, Chamberlain, Joel R., Valdmanis, Paul N.

“…Gene knockdown using short hairpin RNAs (shRNAs) is a promising strategy for targeting dominant mutations; however, delivering too much shRNA can disrupt the…”
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AAV6-mediated Systemic shRNA Delivery Reverses Disease in a Mouse Model of Facioscapulohumeral Muscular Dystrophy by Bortolanza, Sergia, Nonis, Alessandro, Sanvito, Francesca, Maciotta, Simona, Sitia, Giovanni, Wei, Jessica, Torrente, Yvan, Di Serio, Clelia, Chamberlain, Joel R, Gabellini, Davide

“…Treatment of dominantly inherited muscle disorders remains a difficult task considering the need to eliminate the pathogenic gene product in a body-wide…”
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Aging with Muscular Dystrophy: Pathophysiology and Clinical Management by Carter, Gregory T., MD, MS, Weiss, Michael D., MD, Chamberlain, Joel R., PhD, Han, Jay J., MD, Abresch, Richard T., MS, Miró, Jordi, PhD, Jensen, Mark P., PhD

“…Major advances in the fields of medical science and physiology, molecular genetics, biomedical engineering, and computer science have provided individuals with…”
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Gene Targeting of Mutant COL1A2 Alleles in Mesenchymal Stem Cells From Individuals With Osteogenesis Imperfecta by Chamberlain, Joel R, Deyle, David R, Schwarze, Ulrike, Wang, Peirong, Hirata, Roli K, Li, Yi, Byers, Peter H, Russell, David W

“…Mesenchymal stem cells (MSCs) are adult cells with the capacity to differentiate into multiple cell types, including bone, fat, cartilage, and muscle cells. In…”
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Correction: Corrigendum: Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy by 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.

“…Nature Communications 8: Article number: 14454 (2017); Published: 14 February 2017; Updated: 23 June 2017 This Article contains an error in Fig. 4, for which…”
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Gene Targeting in Stem Cells from Individuals with Osteogenesis Imperfecta by Chamberlain, Joel R., Schwarze, Ulrike, Wang, Pei-Rong, Hirata, Roli K., Hankenson, Kurt D., Pace, James M., Underwood, Robert A., Song, Kit M., Sussman, Michael, Byers, Peter H., Russell, David W.

“…Adult stem cells offer the potential to treat many diseases through a combination of ex vivo genetic manipulation and autologous transplantation. Mes-enchymal…”
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Muscling in: Gene therapies for muscular dystrophy target RNA by Chamberlain, Joel R, Chamberlain, Jeffrey S

“…Muscle diseases can take many forms, from the progressive muscle degeneration of dystrophies to the childhood cancer rhabdomyosarcoma. In 'Bench to Bedside',…”
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Therapy for neuromuscular disorders by Arnett, Andrea LH, Chamberlain, Joel R, Chamberlain, Jeffrey S

“…Research into therapeutic approaches for both recessive and dominant neuromuscular disorders has made great progress over the past few years. In the field of…”
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AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy by Bortolanza, Sergia, Nonis, Alessandro, Sanvito, Francesca, Maciotta, Simona, Sitia, Giovanni, Wei, Jessica, Torrente, Yvan, Di Serio, Clelia, Chamberlain, Joel R, Gabellini, Davide

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On fixing broken muscle...fall seven times, stand up eight-Japanese proverb by Carter, Gregory T., Han, Jay J., Chamberlain, Joel R., Chamberlain, Jeffrey S.

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Animal Models of Muscular Dystrophy by Ng, Rainer, Banks, Glen B., Hall, John K., Muir, Lindsey A., Ramos, Julian N., Wicki, Jacqueline, Odom, Guy L., Konieczny, Patryk, Seto, Jane, Chamberlain, Joel R., Chamberlain, Jeffrey S.

“…The muscular dystrophies (MDs) represent a diverse collection of inherited human disorders, which affect to varying degrees skeletal, cardiac, and sometimes…”
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5′ Processing of tRNA Precursors Can Be Modulated by the Human La Antigen Phosphoprotein by Fan, Hao, Goodier, John L., Chamberlain, Joel R., Engelke, David R., Maraia, Richard J.

“…Article Usage Stats Services MCB Citing Articles Google Scholar PubMed Related Content Social Bookmarking CiteULike Delicious Digg Facebook Google+ Mendeley…”
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Chimeric Ad5 Vectors Expressing the Short Fiber of Ad41 Show Reduced Affinity for Human Intestinal Epithelium by Kesisoglou, Filippos, Chamberlain, Joel R, Schmiedlin-Ren, Phyllissa, Kaz, Andrew, Fleisher, David, Roessler, Blake, Zimmermann, Ellen M

“…Altering adenovirus tropism has attracted increased attention in recent years to improve gene delivery. We constructed a recombinant Ad5 vector carrying the…”
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