Molecular and cellular basis of genetically inherited skeletal muscle disorders
Dowling, James J., Weihl, Conrad C., Spencer, Melissa J.
Published in Nature reviews. Molecular cell biology (01.11.2021)
Published in Nature reviews. Molecular cell biology (01.11.2021)
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A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells
Young, Courtney S., Hicks, Michael R., Ermolova, Natalia V., Nakano, Haruko, Jan, Majib, Younesi, Shahab, Karumbayaram, Saravanan, Kumagai-Cresse, Chino, Wang, Derek, Zack, Jerome A., Kohn, Donald B., Nakano, Atsushi, Nelson, Stanley F., Miceli, M. Carrie, Spencer, Melissa J., Pyle, April D.
Published in Cell stem cell (07.04.2016)
Published in Cell stem cell (07.04.2016)
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ERBB3 and NGFR mark a distinct skeletal muscle progenitor cell in human development and hPSCs
Hicks, Michael R., Hiserodt, Julia, Paras, Katrina, Fujiwara, Wakana, Eskin, Ascia, Jan, Majib, Xi, Haibin, Young, Courtney S., Evseenko, Denis, Nelson, Stanley F., Spencer, Melissa J., Handel, Ben Van, Pyle, April D.
Published in Nature cell biology (01.01.2018)
Published in Nature cell biology (01.01.2018)
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Osteopontin ablation ameliorates muscular dystrophy by shifting macrophages to a pro-regenerative phenotype
Capote, Joana, Kramerova, Irina, Martinez, Leonel, Vetrone, Sylvia, Barton, Elisabeth R, Sweeney, H Lee, Miceli, M Carrie, Spencer, Melissa J
Published in The Journal of cell biology (25.04.2016)
Published in The Journal of cell biology (25.04.2016)
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The molecular tweezer CLR01 inhibits aberrant superoxide dismutase 1 (SOD1) self-assembly in vitro and in the G93A-SOD1 mouse model of ALS
Malik, Ravinder, Meng, Helen, Wongkongkathep, Piriya, Corrales, Christian I., Sepanj, Niki, Atlasi, Ryan S., Klärner, Frank-Gerrit, Schrader, Thomas, Spencer, Melissa J., Loo, Joseph A., Wiedau, Martina, Bitan, Gal
Published in The Journal of biological chemistry (08.03.2019)
Published in The Journal of biological chemistry (08.03.2019)
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Genetic modifiers of muscular dystrophy act on sarcolemmal resealing and recovery from injury
Quattrocelli, Mattia, Capote, Joanna, Ohiri, Joyce C, Warner, James L, Vo, Andy H, Earley, Judy U, Hadhazy, Michele, Demonbreun, Alexis R, Spencer, Melissa J, McNally, Elizabeth M
Published in PLoS genetics (24.10.2017)
Published in PLoS genetics (24.10.2017)
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Autolytic Activation of Calpain 3 Proteinase Is Facilitated by Calmodulin Protein
Ermolova, Natalia, Kramerova, Irina, Spencer, Melissa J.
Published in The Journal of biological chemistry (09.01.2015)
Published in The Journal of biological chemistry (09.01.2015)
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Single cell sequencing maps skeletal muscle cellular diversity as disease severity increases in dystrophic mouse models
Saleh, Kholoud K., Xi, Haibin, Switzler, Corey, Skuratovsky, Emily, Romero, Matthew A., Chien, Peggie, Gibbs, Devin, Gane, Lily, Hicks, Michael R., Spencer, Melissa J., Pyle, April D.
Published in iScience (18.11.2022)
Published in iScience (18.11.2022)
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Nanoparticles systemically biodistribute to regenerating skeletal muscle in DMD
Hicks, Michael R, Liu, Xiangsheng, Young, Courtney S, Saleh, Kholoud, Ji, Ying, Jiang, Jinhong, Emami, Michael R, Mokhonova, Ekaterina, Spencer, Melissa J, Meng, Huan, Pyle, April D
Published in Journal of nanobiotechnology (29.08.2023)
Published in Journal of nanobiotechnology (29.08.2023)
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Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy
Young, Courtney S., Mokhonova, Ekaterina, Quinonez, Marbella, Pyle, April D., Spencer, Melissa J.
Published in Journal of neuromuscular diseases (2017)
Published in Journal of neuromuscular diseases (2017)
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Single-cell and spatial transcriptomics identify a macrophage population associated with skeletal muscle fibrosis
Coulis, Gerald, Jaime, Diego, Guerrero-Juarez, Christian, Kastenschmidt, Jenna M, Farahat, Philip K, Nguyen, Quy, Pervolarakis, Nicholas, McLinden, Katherine, Thurlow, Lauren, Movahedi, Saba, Hughes, Brandon S, Duarte, Jorge, Sorn, Andrew, Montoya, Elizabeth, Mozaffar, Izza, Dragan, Morgan, Othy, Shivashankar, Joshi, Trupti, Hans, Chetan P, Kimonis, Virginia, MacLean, Adam L, Nie, Qing, Wallace, Lindsay M, Harper, Scott Q, Mozaffar, Tahseen, Hogarth, Marshall W, Bhattacharya, Surajit, Jaiswal, Jyoti K, Golann, David R, Su, Qi, Kessenbrock, Kai, Stec, Michael, Spencer, Melissa J, Zamudio, Jesse R, Villalta, S Armando
Published in Science advances (07.07.2023)
Published in Science advances (07.07.2023)
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Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy
Kendall, Genevieve C, Mokhonova, Ekaterina I, Moran, Miriana, Sejbuk, Natalia E, Wang, Derek W, Silva, Oscar, Wang, Richard T, Martinez, Leonel, Lu, Qi L, Damoiseaux, Robert, Spencer, Melissa J, Nelson, Stanley F, Miceli, M Carrie
Published in Science translational medicine (12.12.2012)
Published in Science translational medicine (12.12.2012)
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A Nitric Oxide Synthase Transgene Ameliorates Muscular Dystrophy in Mdx Mice
Wehling, Michelle, Spencer, Melissa J., Tidball, James G.
Published in The Journal of cell biology (01.10.2001)
Published in The Journal of cell biology (01.10.2001)
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Skeletal muscle Nur77 expression enhances oxidative metabolism and substrate utilization[S]
Chao, Lily C., Wroblewski, Kevin, Ilkayeva, Olga R., Stevens, Robert D., Bain, James, Meyer, Gretchen A., Schenk, Simon, Martinez, Leonel, Vergnes, Laurent, Narkar, Vihang A., Drew, Brian G., Hong, Cynthia, Boyadjian, Rima, Hevener, Andrea L., Evans, Ronald M., Reue, Karen, Spencer, Melissa J., Newgard, Christopher B., Tontonoz, Peter
Published in Journal of lipid research (01.12.2012)
Published in Journal of lipid research (01.12.2012)
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Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy
Emami, Michael R., Espinoza, Alejandro, Young, Courtney S., Ma, Feiyang, Farahat, Philip K., Felgner, Philip L., Chamberlain, Jeffrey S., Xu, Xiangmin, Pyle, April D., Pellegrini, Matteo, Villalta, S. Armando, Spencer, Melissa J.
Published in Molecular therapy. Methods & clinical development (14.09.2023)
Published in Molecular therapy. Methods & clinical development (14.09.2023)
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Pathogenity of some limb girdle muscular dystrophy mutations can result from reduced anchorage to myofibrils and altered stability of calpain 3
Ermolova, Natalia, Kudryashova, Elena, DiFranco, Marino, Vergara, Julio, Kramerova, Irina, Spencer, Melissa J.
Published in Human molecular genetics (01.09.2011)
Published in Human molecular genetics (01.09.2011)
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Molecular and cellular basis of calpainopathy (limb girdle muscular dystrophy type 2A)
Kramerova, Irina, Beckmann, Jacques S., Spencer, Melissa J.
Published in Biochimica et biophysica acta (01.02.2007)
Published in Biochimica et biophysica acta (01.02.2007)
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A Small-Molecule Approach to Restore a Slow-Oxidative Phenotype and Defective CaMKIIβ Signaling in Limb Girdle Muscular Dystrophy
Liu, Jian, Campagna, Jesus, John, Varghese, Damoiseaux, Robert, Mokhonova, Ekaterina, Becerra, Diana, Meng, Huan, McNally, Elizabeth M., Pyle, April D., Kramerova, Irina, Spencer, Melissa J.
Published in Cell reports. Medicine (20.10.2020)
Published in Cell reports. Medicine (20.10.2020)
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