Search Results - "Martin, Paul T"

Soluble Heparin Binding Epidermal Growth Factor-Like Growth Factor Is a Regulator of GALGT2 Expression and GALGT2-Dependent Muscle and Neuromuscular Phenotypes by Cramer, Megan L., Xu, Rui, Martin, Paul T.

“…GALGT2 (also B4GALNT2) encodes a glycosyltransferase that is normally confined to the neuromuscular and myotendinous junction in adult skeletal muscle. GALGT2…”
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Loss of CMAH during Human Evolution Primed the Monocyte-Macrophage Lineage toward a More Inflammatory and Phagocytic State by Okerblom, Jonathan J, Schwarz, Flavio, Olson, Josh, Fletes, William, Ali, Syed Raza, Martin, Paul T, Glass, Christopher K, Nizet, Victor, Varki, Ajit

“…Humans and chimpanzees are more sensitive to endotoxin than are mice or monkeys, but any underlying differences in inflammatory physiology have not been fully…”
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Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors by Haidet, Amanda M, Rizo, Liza, Handy, Chalonda, Umapathi, Priya, Eagle, Amy, Shilling, Chris, Boue, Daniel, Martin, Paul T, Sahenk, Zarife, Mendell, Jerry R, Kaspar, Brian K

“…Increasing the size and strength of muscles represents a promising therapeutic strategy for musculoskeletal disorders, and interest has focused on myostatin, a…”
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Micro-laminin gene therapy can function as an inhibitor of muscle disease in the dyW mouse model of MDC1A by Packer, Davin, Martin, Paul T.

“…Gene replacement for laminin-α2-deficient congenital muscular dystrophy 1A (MDC1A) is currently not possible using a single adeno-associated virus (AAV) vector…”
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Short-term treatment of golden retriever muscular dystrophy (GRMD) dogs with rAAVrh74.MHCK7.GALGT2 induces muscle glycosylation and utrophin expression but has no significant effect on muscle strength by Martin, Paul T, Zygmunt, Deborah A, Ashbrook, Anna, Hamilton, Sonia, Packer, Davin, Birch, Sharla M, Bettis, Amanda K, Balog-Alvarez, Cynthia J, Guo, Lee-Jae, Nghiem, Peter P, Kornegay, Joe N

“…We have examined the effects of intravenous (IV) delivery of rAAVrh74.MHCK7.GALGT2 in the golden retriever muscular dystrophy (GRMD) model of Duchenne Muscular…”
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Therapeutic efficacy of rscAAVrh74.miniCMV.LIPA gene therapy in a mouse model of lysosomal acid lipase deficiency by Lam, Patricia, Ashbrook, Anna, Zygmunt, Deborah A., Yan, Cong, Du, Hong, Martin, Paul T.

“…Lysosomal acid lipase deficiency (LAL-D) presents as one of two rare autosomal recessive diseases: Wolman disease (WD), a severe disorder presenting in infancy…”
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Vascular Delivery of rAAVrh74.MCK.GALGT2 to the Gastrocnemius Muscle of the Rhesus Macaque Stimulates the Expression of Dystrophin and Laminin α2 Surrogates by Chicoine, Louis G., Rodino-Klapac, Louise R., Shao, Guohong, Xu, Rui, Bremer, William G., Camboni, Marybeth, Golden, Bethannie, Montgomery, Chrystal L., Shontz, Kimberly, Heller, Kristin N., Griffin, Danielle A., Lewis, Sarah, Coley, Brian D., Walker, Christopher M., Clark, K. Reed, Sahenk, Zarife, Mendell, Jerry R., Martin, Paul T.

“…Overexpression of GALGT2 in skeletal muscle can stimulate the glycosylation of α dystroglycan and the upregulation of normally synaptic dystroglycan-binding…”
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Sarcospan-dependent Akt activation is required for utrophin expression and muscle regeneration by Marshall, Jamie L, Holmberg, Johan, Chou, Eric, Ocampo, Amber C, Oh, Jennifer, Lee, Joy, Peter, Angela K, Martin, Paul T, Crosbie-Watson, Rachelle H

“…Utrophin is normally confined to the neuromuscular junction (NMJ) in adult muscle and partially compensates for the loss of dystrophin in mdx mice. We show…”
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Expansion of B4GALT7 linkeropathy phenotype to include perinatal lethal skeletal dysplasia by Mihalic Mosher, Theresa, Zygmunt, Deborah A, Koboldt, Daniel C, Kelly, Benjamin J, Johnson, Lisa R, McKenna, David S, Hood, Benjamin C, Hickey, Scott E, White, Peter, Wilson, Richard K, Martin, Paul T, McBride, Kim L

“…Proteoglycans have a core polypeptide connected to glycosaminoglycans (GAGs) via a common tetrasaccharide linker region. Defects in enzymes that synthesize the…”
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Identification of new dystroglycan complexes in skeletal muscle by Johnson, Eric K, Li, Bin, Yoon, Jung Hae, Flanigan, Kevin M, Martin, Paul T, Ervasti, James, Montanaro, Federica

“…The dystroglycan complex contains the transmembrane protein β-dystroglycan and its interacting extracellular mucin-like protein α-dystroglycan. In skeletal…”
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Deletion of Pofut1 in Mouse Skeletal Myofibers Induces Muscle Aging-Related Phenotypes in cis and in trans by Zygmunt, Deborah A., Singhal, Neha, Kim, Mi-Lyang, Cramer, Megan L., Crowe, Kelly E., Xu, Rui, Jia, Ying, Adair, Jessica, Martinez-Pena y Valenzuela, Isabel, Akaaboune, Mohammed, White, Peter, Janssen, Paulus M., Martin, Paul T.

“…Sarcopenia, the loss of muscle mass and strength during normal aging, involves coordinate changes in skeletal myofibers and the cells that contact them,…”
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A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2 by Flanigan, Kevin M., Vetter, Tatyana A., Simmons, Tabatha R., Iammarino, Megan, Frair, Emma C., Rinaldi, Federica, Chicoine, Louis G., Harris, Johan, Cheatham, John P., Cheatham, Sharon L., Boe, Brian, Waldrop, Megan A., Zygmunt, Deborah A., Packer, Davin, Martin, Paul T.

“…In a phase 1/2, open-label dose escalation trial, we delivered rAAVrh74.MCK.GALGT2 (also B4GALNT2) bilaterally to the legs of two boys with Duchenne muscular…”
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Induction of a regenerative microenvironment in skeletal muscle is sufficient to induce embryonal rhabdomyosarcoma in p53-deficient mice by Camboni, Marybeth, Hammond, Sue, Martin, Laura T, Martin, Paul T

“…We have previously reported that mice with muscular dystrophy, including mdx mice, develop embryonal rhabdomyosarcoma (eRMS) with a low incidence after 1 year…”
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rAAVrh74.MCK.GALGT2 Demonstrates Safety and Widespread Muscle Glycosylation after Intravenous Delivery in C57BL/6J Mice by Zygmunt, Deborah A., Xu, Rui, Jia, Ying, Ashbrook, Anna, Menke, Chelsea, Shao, Guohong, Yoon, Jung Hae, Hamilton, Sonia, Pisharath, Harshan, Bolon, Brad, Martin, Paul T.

“…rAAVrh74.MCK.GALGT2 is a surrogate gene therapy that inhibits muscular dystrophy in multiple animal models. Here, we report on a dose-response study of…”
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A comparative study of N-glycolylneuraminic acid (Neu5Gc) and cytotoxic T cell (CT) carbohydrate expression in normal and dystrophin-deficient dog and human skeletal muscle by Martin, Paul T, Golden, Bethannie, Okerblom, Jonathan, Camboni, Marybeth, Chandrasekharan, Kumaran, Xu, Rui, Varki, Ajit, Flanigan, Kevin M, Kornegay, Joe N

“…The expression of N-glycolylneuraminic acid (Neu5Gc) and the cytotoxic T cell (CT) carbohydrate can impact the severity of muscular dystrophy arising from the…”
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The Dystroglycanopathies: The New Disorders of O-Linked Glycosylation by Martin, Paul T.

“…It has become clear in the past half decade that a number of forms of congenital muscular dystrophy are in fact congenital disorders of glycosylation. Genes…”
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Role of extracellular matrix proteins and their receptors in the development of the vertebrate neuromuscular junction by Singhal, Neha, Martin, Paul T.

“…The vertebrate neuromuscular junction (NMJ) remains the best‐studied model for understanding the mechanisms involved in synaptogenesis, due to its relatively…”
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A lethal injection? by Arumugam, Deepak, Dr, Atherton, John J, PhD, Martin, Paul T, PhD

“…Procainamide, which is similar to procaine (webappendix), is an antiarrhythmic drug that causes sodium channel blockade and should be avoided in patients with…”
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Active and passive immunization strategies based on the SDPM1 peptide demonstrate pre-clinical efficacy in the APPswePSEN1dE9 mouse model for Alzheimer's disease by Camboni, Marybeth, Wang, Chiou-Miin, Miranda, Carlos, Yoon, Jung Hae, Xu, Rui, Zygmunt, Deborah, Kaspar, Brian K, Martin, Paul T

“…Abstract Recent clinical and pre-clinical studies suggest that both active and passive immunization strategies targeting Aβ amyloid may have clinical benefit…”
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rAAVrh74.MCK.GALGT2 Protects against Loss of Hemodynamic Function in the Aging mdx Mouse Heart by Xu, Rui, Jia, Ying, Zygmunt, Deborah A., Martin, Paul T.

“…Dilated cardiomyopathy is a common cause of death in patients with Duchenne muscular dystrophy (DMD). Gene therapies for DMD must, therefore, have a…”
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