Targeting nuclear RNA for in vivo correction of myotonic dystrophy von Wheeler, Thurman M., Leger, Andrew J., Pandey, Sanjay K., MacLeod, A. Robert, Nakamori, Masayuki, Cheng, Seng H., Wentworth, Bruce M., Bennett, C. Frank, Thornton, Charles A.

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Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics von Chen, Jennifer CJ, King, Oliver D, Zhang, Yuanfan, Clayton, Nicholas P, Spencer, Carrie, Wentworth, Bruce M, Emerson, Charles P, Wagner, Kathryn R

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A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice von Gan, Li, Wu, Leslie C.L., Wood, Jenna A., Yao, Monica, Treleaven, Chris M., Estrella, Nelsa L., Wentworth, Bruce M., Hanson, Gunnar J., Passini, Marco A.

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Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease von Clayton, Nicholas P, Nelson, Carol A, Weeden, Timothy, Taylor, Kristin M, Moreland, Rodney J, Scheule, Ronald K, Phillips, Lucy, Leger, Andrew J, Cheng, Seng H, Wentworth, Bruce M

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Dimorphic effects of transforming growth factor-β signaling during aortic aneurysm progression in mice suggest a combinatorial therapy for Marfan syndrome von Cook, Jason R, Clayton, Nicholas P, Carta, Luca, Galatioto, Josephine, Chiu, Emily, Smaldone, Silvia, Nelson, Carol A, Cheng, Seng H, Wentworth, Bruce M, Ramirez, Francesco

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Fibrillin‐1 Regulates Skeletal Stem Cell Differentiation by Modulating TGFβ Activity Within the Marrow Niche von Smaldone, Silvia, Clayton, Nicholas P, del Solar, Maria, Pascual, Gemma, Cheng, Seng H, Wentworth, Bruce M, Schaffler, Mitchell B, Ramirez, Francesco

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Inhibiting TGF-β Activity Improves Respiratory Function in mdx Mice von Nelson, Carol A, Hunter, R. Bridge, Quigley, Lindsay A, Girgenrath, Stefan, Weber, William D, McCullough, Jennifer A, Dinardo, Carol J, Keefe, Kelly A, Ceci, Lorena, Clayton, Nicholas P, McVie-Wylie, Alison, Cheng, Seng H, Leonard, John P, Wentworth, Bruce M

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Systemic Delivery of a Peptide-Linked Morpholino Oligonucleotide Neutralizes Mutant RNA Toxicity in a Mouse Model of Myotonic Dystrophy von Leger, Andrew J., Mosquea, Leocadia M., Clayton, Nicholas P., Wu, I-Huan, Weeden, Timothy, Nelson, Carol A., Phillips, Lucy, Roberts, Errin, Piepenhagen, Peter A., Cheng, Seng H., Wentworth, Bruce M.

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Systemic Delivery of a Peptide-Linked Morpholino Oligonucleotide Neutralizes Mutant RNA Toxicity in a Mouse Model of Myotonic Dystrophy von Leger, Andrew J., Mosquea, Leocadia M., Clayton, Nicholas P., Wu, I-Huan, Weeden, Timothy, Nelson, Carol A., Phillips, Lucy, Roberts, Errin, Piepenhagen, Peter A., Cheng, Seng H., Wentworth, Bruce M.

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Development of PPMO for the Treatment of Duchenne Muscular Dystrophy (P5.430) von Passini, Marco A., Gan, Li, Wood, Jenna A., Yao, Monica, Estrella, Nelsa L., Treleaven, Chris M., Wentworth, Bruce M., Charleston, Jay S., Rutkowski, Joseph V., Hanson, Gunnar J.

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Fibrillin-1 Regulates Skeletal Stem Cell Differentiation by Modulating TGF[beta] Activity Within the Marrow Niche von Smaldone, Silvia, Clayton, Nicholas P, del Solar, Maria, Pascual, Gemma, Cheng, Seng H, Wentworth, Bruce M, Schaffler, Mitchell B, Ramirez, Francesco

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Dimorphic effects of TGFβ signaling during aortic aneurysm progression in mice suggest a combinatorial therapy for Marfan syndrome von Cook, Jason R., Clayton, Nicholas P., Carta, Luca, Galatioto, Josephine, Chiu, Emily, Smaldone, Silvia, Nelson, Carol A., Cheng, Seng H., Wentworth, Bruce M., Ramirez, Francesco

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Antisense oligonucleotide-mediated suppression of muscle glycogen synthase 1 synthesis as an approach for substrate reduction therapy of Pompe disease von Clayton, Nicholas P., Nelson, Carol A., Weeden, Timothy, Taylor, Kristin M., Moreland, Rodney J., Scheule, Ronald K., Leger, Andrew J., Phillips, Lucy, Cheng, Seng H., Wentworth, Bruce M.

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A highly conserved enhancer downstream of the human MLC1/3 locus is a target for multiple myogenic determination factors von ROSENTHAL, N, BERGLUND, E. B, WENTWORTH, B. M, DONOGHUE, M, WINTER, B, BOBER, E, BRAUN, T, ARNOLD, H.-H

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy von Wheeler, Thurman M, Leger, Andrew J, Pandey, Sanjay K, MacLeod, A Robert, Nakamori, Masayuki, Cheng, Seng H, Wentworth, Bruce M, Bennett, C Frank, Thornton, Charles A

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Characterization of the two nonallelic genes encoding mouse preproinsulin von Wentworth, B M, Schaefer, I M, Villa-Komaroff, L, Chirgwin, J M

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Characterization of proliferating human skeletal muscle-derived cells in vitro: Differential modulation of myoblast markers by TGF-β2 von Stewart, Jeffrey D., Masi, Terése L., Cumming, Andrew E., Molnar, Gyongyi M., Wentworth, Bruce M., Sampath, Kuber, McPherson, John M., Yaeger, Peter C.

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Alternative delivery of keratinocytes using a polyurethane membrane and the implications for its use in the treatment of full-thickness burn injury von Wright, Kathryn A., Nadire, Kathleen B., Busto, Patricia, Tubo, Ross, McPherson, John M., Wentworth, Bruce M.

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Paired MyoD-Binding Sites Regulate Myosin Light Chain Gene Expression von Wentworth, Bruce M., Donoghue, Maria, Engert, James C., Berglund, Erick B., Rosenthal, Nadia

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Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides von Wentworth, Bruce M, Nelson, Carol A, Clayton, Nicholas P, Scheule, Ronald K, Weeden, Timothy E

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