Eteplirsen for the treatment of Duchenne muscular dystrophy
Mendell, Jerry R., Rodino-Klapac, Louise R., Sahenk, Zarife, Roush, Kandice, Bird, Loren, Lowes, Linda P., Alfano, Lindsay, Gomez, Ann Maria, Lewis, Sarah, Kota, Janaiah, Malik, Vinod, Shontz, Kim, Walker, Christopher M., Flanigan, Kevin M., Corridore, Marco, Kean, John R., Allen, Hugh D., Shilling, Chris, Melia, Kathleen R., Sazani, Peter, Saoud, Jay B., Kaye, Edward M.
Published in Annals of neurology (01.11.2013)
Published in Annals of neurology (01.11.2013)
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Journal Article
A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy
Mendell, Jerry R, Sahenk, Zarife, Malik, Vinod, Gomez, Ana M, Flanigan, Kevin M, Lowes, Linda P, Alfano, Lindsay N, Berry, Katherine, Meadows, Eric, Lewis, Sarah, Braun, Lyndsey, Shontz, Kim, Rouhana, Maria, Clark, Kelly Reed, Rosales, Xiomara Q, Al-Zaidy, Samiah, Govoni, Alessandra, Rodino-Klapac, Louise R, Hogan, Mark J, Kaspar, Brian K
Published in Molecular therapy (01.01.2015)
Published in Molecular therapy (01.01.2015)
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Journal Article
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes
Mendell, Jerry R., Sahenk, Zarife, Al-Zaidy, Samiah, Rodino-Klapac, Louise R., Lowes, Linda P., Alfano, Lindsay N., Berry, Katherine, Miller, Natalie, Yalvac, Mehmet, Dvorchik, Igor, Moore-Clingenpeel, Melissa, Flanigan, Kevin M., Church, Kathleen, Shontz, Kim, Curry, Choumpree, Lewis, Sarah, McColly, Markus, Hogan, Mark J., Kaspar, Brian K.
Published in Molecular therapy (05.04.2017)
Published in Molecular therapy (05.04.2017)
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Journal Article
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models
Sondergaard, Patricia C., Griffin, Danielle A., Pozsgai, Eric R., Johnson, Ryan W., Grose, William E., Heller, Kristin N., Shontz, Kim M., Montgomery, Chrystal L., Liu, Joseph, Clark, Kelly Reed, Sahenk, Zarife, Mendell, Jerry R., Rodino‐Klapac, Louise R.
Published in Annals of clinical and translational neurology (01.03.2015)
Published in Annals of clinical and translational neurology (01.03.2015)
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Journal Article
Safety and Efficacy of AAV Retrograde Pancreatic Ductal Gene Delivery in Normal and Pancreatic Cancer Mice
Quirin, Kayla A, Kwon, Jason J, Alioufi, Arafat, Factora, Tricia, Temm, Constance J, Jacobsen, Max, Sandusky, George E, Shontz, Kim, Chicoine, Louis G, Clark, K Reed, Mendell, Joshua T, Korc, Murray, Kota, Janaiah
Published in Molecular therapy. Methods & clinical development (16.03.2018)
Published in Molecular therapy. Methods & clinical development (16.03.2018)
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Journal Article
609. Systemic Delivery of Dysferlin Overlap Vectors Mediates Functional Recovery of Dysferlin Deficiency
Sondergaard, Patricia C., Griffin, Danielle A., Pozsgai, Eric R., Johnson, Ryan, Shontz, Kim, Mendell, Jerry R., Rodmo-Klapac, Louise R.
Published in Molecular therapy (01.05.2015)
Published in Molecular therapy (01.05.2015)
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Journal Article
Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice
Martin, Paul T, Xu, Rui, Rodino-Klapac, Louise R, Oglesbay, Elaine, Camboni, Marybeth, Montgomery, Chrystal L, Shontz, Kim, Chicoine, Louis G, Clark, K. Reed, Sahenk, Zarife, Mendell, Jerry R, Janssen, Paul M. L
Published in American Journal of Physiology: Cell Physiology (01.03.2009)
Published in American Journal of Physiology: Cell Physiology (01.03.2009)
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Journal Article
C-2. Eteplirsen, a Phosphorodiamidate Morpholino Oligomer (PMO) for the Treatment of Duchenne Muscular Dystrophy (DMD): 168 Week Update on Six-Minute Walk Test (6MWT), Pulmonary Function Testing (PFT), and Safety
Mendell, Jerry R., Rodino-Klapac, Louise, Sahenk, Zarife, Rouch, Kandice, Bird, Lauren, Lowes, Linda, Alfano, Lindsay, Berry, Katherine, Lewis, Sarah, Shontz, Kim, Flanigan, Kevin, Shilling, Christopher, Duda, Petra, Saoud, Jay
Published in Molecular therapy (01.05.2015)
Published in Molecular therapy (01.05.2015)
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Journal Article
A Potential microRNA-Based Therapy for Pancreatic Ductal Adenocarcinoma Using AAV-Mediated Delivery
Kota, Janaiah, Chivukula, Raghu R, Kent, Oliver A, Shontz, Kim M, Maitra, Anirban, Clark, K Reed, Mendell, Jerry R, Mendell, Joshua T
Published in Molecular therapy (01.07.2011)
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Published in Molecular therapy (01.07.2011)
Journal Article
Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice
MARTIN, Paul T, RUI XU, MENDELL, Jerry R, JANSSEN, Paul M. L, RODINO-KLAPAC, Louise R, OGLESBAY, Elaine, CAMBONI, Marybeth, MONTGOMERY, Chrystal L, SHONTZ, Kim, CHICOINE, Louis G, CLARK, K. Reed, SAHENK, Zarife
Published in American Journal of Physiology: Cell Physiology (2009)
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Published in American Journal of Physiology: Cell Physiology (2009)
Journal Article