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)
Get full text
Journal Article
High levels of AAV vector integration into CRISPR-induced DNA breaks
Hanlon, Killian S., Kleinstiver, Benjamin P., Garcia, Sara P., Zaborowski, Mikołaj P., Volak, Adrienn, Spirig, Stefan E., Muller, Alissa, Sousa, Alexander A., Tsai, Shengdar Q., Bengtsson, Niclas E., Lööv, Camilla, Ingelsson, Martin, Chamberlain, Jeffrey S., Corey, David P., Aryee, Martin J., Joung, J. Keith, Breakefield, Xandra O., Maguire, Casey A., György, Bence
Published in Nature communications (30.09.2019)
Published in Nature communications (30.09.2019)
Get full text
Journal Article
Gene and cell-mediated therapies for muscular dystrophy
Konieczny, Patryk, Swiderski, Kristy, Chamberlain, Jeffrey S.
Published in Muscle & nerve (01.05.2013)
Published in Muscle & nerve (01.05.2013)
Get full text
Journal 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
Lai, Yi, Thomas, Gail D, Yue, Yongping, Yang, Hsiao T, Li, Dejia, Long, Chun, Judge, Luke, Bostick, Brian, Chamberlain, Jeffrey S, Terjung, Ronald L, Duan, Dongsheng
Published in The Journal of clinical investigation (01.03.2009)
Published in The Journal of clinical investigation (01.03.2009)
Get full text
Journal Article
Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials
Chamberlain, Jeffrey S, Robb, Melissa, Braun, Serge, Brown, Kristy J, Danos, Olivier, Ganot, Annie, Gonzalez-Alegre, Pedro, Hunter, Nina, McDonald, Craig, Morris, Carl, Tobolowsky, Mark, Wagner, Kathryn R, Ziolkowski, Olivia, Duan, Dongsheng
Published in Human gene therapy (01.05.2023)
Published in Human gene therapy (01.05.2023)
Get more information
Journal Article
Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells
Bianco, Paolo, Cossu, Giulio, Dellavalle, Arianna, Sampaolesi, Maurilio, Tonlorenzi1, Rossana, Tagliafico, Enrico, Sacchetti, Benedetto, Perani, Laura, Innocenzi, Anna, Galvez, Beatriz G, Messina, Graziella, Morosetti, Roberta, Li, Sheng, Belicchi, Marzia, Peretti, Giuseppe, Chamberlain, Jeffrey S, Wright, Woodring E, Torrente, Yvan, Ferrari, Stefano
Published in Nature cell biology (01.03.2007)
Published in Nature cell biology (01.03.2007)
Get full text
Journal Article
Follistatin-mediated skeletal muscle hypertrophy is regulated by Smad3 and mTOR independently of myostatin
Winbanks, Catherine E, Weeks, Kate L, Thomson, Rachel E, Sepulveda, Patricio V, Beyer, Claudia, Qian, Hongwei, Chen, Justin L, Allen, James M, Lancaster, Graeme I, Febbraio, Mark A, Harrison, Craig A, McMullen, Julie R, Chamberlain, Jeffrey S, Gregorevic, Paul
Published in The Journal of cell biology (25.06.2012)
Published in The Journal of cell biology (25.06.2012)
Get full text
Journal Article
An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells
Filareto, Antonio, Parker, Sarah, Darabi, Radbod, Borges, Luciene, Iacovino, Michelina, Schaaf, Tory, Mayerhofer, Timothy, Chamberlain, Jeffrey S., Ervasti, James M., McIvor, R. Scott, Kyba, Michael, Perlingeiro, Rita C. R.
Published in Nature communications (2013)
Published in Nature communications (2013)
Get full text
Journal Article
Sarcolemma-localized nNOS is required to maintain activity after mild exercise
Campbell, Kevin P, Kobayashi, Yvonne M, Rader, Erik P, Crawford, Robert W, Iyengar, Nikhil K, Thedens, Daniel R, Faulkner, John A, Parikh, Swapnesh V, Weiss, Robert M, Chamberlain, Jeffrey S, Moore, Steven A
Published in Nature (27.11.2008)
Published in Nature (27.11.2008)
Get full text
Journal Article
Prevention of muscle aging by myofiber-associated satellite cell transplantation
Hall, John K, Banks, Glen B, Chamberlain, Jeffrey S, Olwin, Bradley B
Published in Science translational medicine (10.11.2010)
Published in Science translational medicine (10.11.2010)
Get more information
Journal Article
rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice
Chamberlain, Jeffrey S, Gregorevic, Paul, Allen, James M, Minami, Elina, Blankinship, Michael J, Haraguchi, Miki, Meuse, Leonard, Finn, Eric, Adams, Marvin E, Froehner, Stanley C, Murry, Charles E
Published in Nature medicine (01.07.2006)
Published in Nature medicine (01.07.2006)
Get full text
Journal Article
Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy
Ho, Peggy P., Lahey, Lauren J., Mourkioti, Foteini, Kraft, Peggy E., Filareto, Antonio, Brandt, Moritz, Magnusson, Klas E. G., Finn, Eric E., Chamberlain, Jeffrey S., Robinson, William H., Blau, Helen M., Steinman, Lawrence
Published in Proceedings of the National Academy of Sciences - PNAS (25.09.2018)
Published in Proceedings of the National Academy of Sciences - PNAS (25.09.2018)
Get full text
Journal Article
Successful Regional Delivery and Long-term Expression of a Dystrophin Gene in Canine Muscular Dystrophy: A Preclinical Model for Human Therapies
Wang, Zejing, Storb, Rainer, Halbert, Christine L, Banks, Glen B., Butts, Tiffany M., Finn, Eric E., Allen, James M., Miller, A. Dusty, Chamberlain, Jeffrey S., Tapscott, Stephen J.
Published in Molecular therapy (01.08.2012)
Published in Molecular therapy (01.08.2012)
Get full text
Journal Article