Search Results - "Donsante, Anthony"

AAV Vector Integration Sites in Mouse Hepatocellular Carcinoma by Donsante, Anthony, Miller, Daniel G, Li, Yi, Vogler, Carole, Brunt, Elizabeth M, Russell, David W, Sands, Mark S

“…Adeno-associated viruses (AAV) are promising gene therapy vectors that have little or no acute toxicity. We show that normal mice and mice with…”
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A novel antibody to treat SOD1-related amyotrophic lateral sclerosis by Boulis, Nicholas M., Donsante, Anthony

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Lentiviral-Induced Spinal Cord Gliomas in Rat Model by Nagarajan, Purva P, Tora, Muhibullah S, Neill, Stewart G, Federici, Thais, Texakalidis, Pavlos, Donsante, Anthony, Canoll, Peter, Lei, Kecheng, Boulis, Nicholas M

“…Intramedullary spinal cord tumors are a rare and understudied cancer with poor treatment options and prognosis. Our prior study used a combination of PDGF-B,…”
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ATP7A Gene Addition to the Choroid Plexus Results in Long-term Rescue of the Lethal Copper Transport Defect in a Menkes Disease Mouse Model by Donsante, Anthony, Yi, Ling, Zerfas, Patricia M, Brinster, Lauren R, Sullivan, Patricia, Goldstein, David S, Prohaska, Joseph, Centeno, Jose A, Rushing, Elisabeth, Kaler, Stephen G

“…Menkes disease is a lethal infantile neurodegenerative disorder of copper metabolism caused by mutations in a P-type ATPase, ATP7A. Currently available…”
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Altered intracellular localization and valosin-containing protein (p97 VCP) interaction underlie ATP7A-related distal motor neuropathy by LING YI, DONSANTE, Anthony, KENNERSON, Marina L, MERCER, Julian F. B, GARBERN, James Y, KALER, Stephen G

“…ATP7A is a P-type ATPase that regulates cellular copper homeostasis by activity at the trans-Golgi network (TGN) and plasma membrane (PM), with the location…”
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Molecular correlates of epilepsy in early diagnosed and treated Menkes disease by Kaler, Stephen G, Liew, Clarissa J, Donsante, Anthony, Hicks, Julia D, Sato, Susumu, Greenfield, Jacquelyn C

“…Epilepsy is a major feature of Menkes disease, an X-linked recessive infantile neurodegenerative disorder caused by mutations in ATP7A, which produces a…”
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Translational read-through of a nonsense mutation in ATP7A impacts treatment outcome in Menkes disease by Kaler, Stephen G., Tang, Jingrong, Donsante, Anthony, Kaneski, Christine R.

“…Protein translation ends when a stop codon in a gene's messenger RNA transcript enters the ribosomal A site. Mutations that create premature stop codons…”
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Gene, Stem Cell, and Alternative Therapies for SCA 1 by Wagner, Jacob L, O'Connor, Deirdre M, Donsante, Anthony, Boulis, Nicholas M

“…Spinocerebellar ataxia 1 is an autosomal dominant disease characterized by neurodegeneration and motor dysfunction. In disease pathogenesis, polyglutamine…”
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Fetal Brain-directed AAV Gene Therapy Results in Rapid, Robust, and Persistent Transduction of Mouse Choroid Plexus Epithelia by Haddad, Marie Reine, Donsante, Anthony, Zerfas, Patricia, Kaler, Stephen G

“…Fetal brain-directed gene addition represents an under-appreciated tool for investigating novel therapeutic approaches in animal models of central nervous…”
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Molecular and biochemical characterization of a unique mutation in CCS, the human copper chaperone to superoxide dismutase by Huppke, Peter, Brendel, Cornelia, Korenke, Georg Christoph, Marquardt, Iris, Donsante, Anthony, Yi, Ling, Hicks, Julia D., Steinbach, Peter J., Wilson, Callum, Elpeleg, Orly, Møller, Lisbeth Birk, Christodoulou, John, Kaler, Stephen G., Gärtner, Jutta

“…Copper (Cu) is a trace metal that readily gains and donates electrons, a property that renders it desirable as an enzyme cofactor but dangerous as a source of…”
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Molecular and Cellular Therapies for Motor Neuron Diseases by Boulis, Nicholas M, O'Connor, Deirdre, Donsante, Anthony

“…Motor Neuron Diseases (MND) are a group of neurological disorders that affect motor neurons in adults and in children. The fields of gene and cell therapies…”
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Progress in gene and cell therapies for the neuronal ceroid lipofuscinoses by Donsante, Anthony, Boulis, Nicholas M

“…The neuronal ceroid lipofuscinoses (NCLs) are a subset of lysosomal storage diseases (LSDs) that cause myoclonic epilepsy, loss of cognitive and motor…”
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TMOD-23. LENTIVIRAL-INDUCED BRAIN GLIOMAS IN RAT MODEL by Zhang, Michelle, Lakhina, Yuliya, Yonk, Marybeth, Poth, Kelly, Neill, Stewart, Zhang, Zhaobin, Assed, Hemza, Tora, Muhibullah, Donsante, Anthony, Federici, Thais, Lei, Kecheng, Boulis, Nicholas

“…Abstract High-grade gliomas are the most common type of malignant intracranial tumor with dismal five-year prognostic outcomes despite developments in…”
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Minimally Invasive Injection to the Phrenic Nerve in a Porcine Hemidiaphragmatic Paralysis Model: A Pilot Study by Texakalidis, Pavlos, Tora, Muhibullah S, Canute, Skyler, Hardcastle, Nathan, Poth, Kelly, Donsante, Anthony, Federici, Thais, Javidfar, Jeffrey, Boulis, Nicholas M

“…Abstract BACKGROUND Neurodegenerative diseases and spinal cord injury can affect respiratory function often through motor neuron loss innervating the…”
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Gene Therapy for the Treatment of Neurological Disorders: Amyotrophic Lateral Sclerosis by McEachin, Zachary T, Donsante, Anthony, Boulis, Nicholas

“…Gene therapy is a powerful tool for treating diseases, including neurological disorder such at amyotrophic lateral sclerosis. When delivered to the CNS, gene…”
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Controlling the Release of Neurotrophin‐3 and Chondroitinase ABC Enhances the Efficacy of Nerve Guidance Conduits by Donsante, Anthony, Xue, Jiajia, Poth, Kelly M., Hardcastle, Nathan S., Diniz, Bruna, O'Connor, Deirdre M., Xia, Younan, Boulis, Nicholas M.

“…Nerve guidance conduits (NGCs) have the potential to replace autografts in repairing peripheral nerve injuries, but their efficacy still needs to be improved…”
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Lot-to-Lot Variation in Adeno-Associated Virus Serotype 9 (AAV9) Preparations by O'Connor, Deirdre M, Lutomski, Corinne, Jarrold, Martin F, Boulis, Nicholas M, Donsante, Anthony

“…Viral vectors are complex drugs that pose a particular challenge for manufacturing. Previous studies have shown that, unlike small-molecule drugs, vector…”
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Central Nervous System-directed AAV2/5-Mediated Gene Therapy Synergizes with Bone Marrow Transplantation in the Murine Model of Globoid-cell Leukodystrophy by Lin, Darshong, Donsante, Anthony, Macauley, Shannon, Levy, Beth, Vogler, Carole, Sands, Mark S

“…Globoid-cell leukodystrophy (GLD) is a rapidly progressing inherited neurodegenerative disorder caused by a deficiency in galactosylceramidase activity…”
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Somatic mosaicism in Menkes disease suggests choroid plexus-mediated copper transport to the developing brain by Donsante, Anthony, Johnson, Paul, Jansen, Laura A., Kaler, Stephen G.

“…The primary mechanism of copper transport to the brain is unknown, although this process is drastically impaired in Menkes disease, an X‐linked…”
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L-Threo-Dihydroxyphenylserine corrects neurochemical abnormalities in a menkes disease mouse model by Donsante, Anthony, Sullivan, Patricia, Goldstein, David S., Brinster, Lauren R., Kaler, Stephen G.

“…Objective Menkes disease is a lethal neurodegenerative disorder of infancy caused by mutations in a copper‐transporting adenosine triphosphatase gene, ATP7A…”
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