Search Results - "Kaspar, Brian"

Macrophage Migration Inhibitory Factor as a Chaperone Inhibiting Accumulation of Misfolded SOD1 by Israelson, Adrian, Ditsworth, Dara, Sun, Shuying, Song, SungWon, Liang, Jason, Hruska-Plochan, Marian, McAlonis-Downes, Melissa, Abu-Hamad, Salah, Zoltsman, Guy, Shani, Tom, Maldonado, Marcus, Bui, Anh, Navarro, Michael, Zhou, Huilin, Marsala, Martin, Kaspar, Brian K., Da Cruz, Sandrine, Cleveland, Don W.

“…Mutations in superoxide dismutase (SOD1) cause amyotrophic lateral sclerosis (ALS), a neurodegenerative disease characterized by loss of motor neurons and…”
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Microglia Induce Motor Neuron Death via the Classical NF-κB Pathway in Amyotrophic Lateral Sclerosis by Frakes, Ashley E., Ferraiuolo, Laura, Haidet-Phillips, Amanda M., Schmelzer, Leah, Braun, Lyndsey, Miranda, Carlos J., Ladner, Katherine J., Bevan, Adam K., Foust, Kevin D., Godbout, Jonathan P., Popovich, Phillip G., Guttridge, Denis C., Kaspar, Brian K.

“…Neuroinflammation is one of the most striking hallmarks of amyotrophic lateral sclerosis (ALS). Nuclear factor-kappa B (NF-κB), a master regulator of…”
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Glia–neuron interactions in neurological diseases: Testing non-cell autonomy in a dish by Meyer, Kathrin, Kaspar, Brian K

“…Abstract For the past century, research on neurological disorders has largely focused on the most prominently affected cell types – the neurons. However, with…”
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Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates by Meyer, Kathrin, Ferraiuolo, Laura, Schmelzer, Leah, Braun, Lyndsey, McGovern, Vicki, Likhite, Shibi, Michels, Olivia, Govoni, Alessandra, Fitzgerald, Julie, Morales, Pablo, Foust, Kevin D, Mendell, Jerry R, Burghes, Arthur H M, Kaspar, Brian K

“…Spinal muscular atrophy (SMA) is the most frequent lethal genetic neurodegenerative disorder in infants. The disease is caused by low abundance of the survival…”
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Translational profiling identifies a cascade of damage initiated in motor neurons and spreading to glia in mutant SOD1-mediated ALS by Sun, Shuying, Sun, Ying, Ling, Shuo-Chien, Ferraiuolo, Laura, McAlonis-Downes, Melissa, Zou, Yiyang, Drenner, Kevin, Wang, Yin, Ditsworth, Dara, Tokunaga, Seiya, Kopelevich, Alex, Kaspar, Brian K., Lagier-Tourenne, Clotilde, Cleveland, Don W.

“…Ubiquitous expression of amyotrophic lateral sclerosis (ALS)-causing mutations in superoxide dismutase 1 (SOD1) provokes noncell autonomous paralytic disease…”
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Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS by Meyer, Kathrin, Ferraiuolo, Laura, Miranda, Carlos J., Likhite, Shibi, McElroy, Sohyun, Renusch, Samantha, Ditsworth, Dara, Lagier-Tourenne, Clotilde, Smith, Richard A., Ravits, John, Burghes, Arthur H., Shaw, Pamela J., Cleveland, Don W., Kolb, Stephen J., Kaspar, Brian K.

“…Amyotrophic lateral sclerosis (ALS) causes motor neuron degeneration, paralysis, and death. Accurate disease modeling, identifying disease mechanisms, and…”
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The C9orf72 protein interacts with Rab1a and the ULK1 complex to regulate initiation of autophagy by Webster, Christopher P, Smith, Emma F, Bauer, Claudia S, Moller, Annekathrin, Hautbergue, Guillaume M, Ferraiuolo, Laura, Myszczynska, Monika A, Higginbottom, Adrian, Walsh, Matthew J, Whitworth, Alexander J, Kaspar, Brian K, Meyer, Kathrin, Shaw, Pamela J, Grierson, Andrew J, De Vos, Kurt J

“…A GGGGCC hexanucleotide repeat expansion in the C9orf72 gene is the most common genetic cause of amyotrophic lateral sclerosis and frontotemporal dementia…”
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Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism by Ferraiuolo, Laura, Meyer, Kathrin, Sherwood, Thomas W., Vick, Jonathan, Likhite, Shibi, Frakes, Ashley, Miranda, Carlos J., Braun, Lyndsey, Heath, Paul R., Pineda, Ricardo, Beattie, Christine E., Shaw, Pamela J., Askwith, Candice C., McTigue, Dana, Kaspar, Brian K.

“…Oligodendrocytes have recently been implicated in the pathophysiology of amyotrophic lateral sclerosis (ALS). Here we show that, in vitro, mutant superoxide…”
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A role for glia in the progression of Rett’s syndrome by Lioy, Daniel T., Garg, Saurabh K., Monaghan, Caitlin E., Raber, Jacob, Foust, Kevin D., Kaspar, Brian K., Hirrlinger, Petra G., Kirchhoff, Frank, Bissonnette, John M., Ballas, Nurit, Mandel, Gail

“…Astrocytes targeted in Rett's syndrome Rett's syndrome, a disorder caused by loss of function of methyl CpG-binding protein 2 (MeCP2), is associated with…”
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Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes by Kaspar, Brian K, Foust, Kevin D, Nurre, Emily, Montgomery, Chrystal L, Hernandez, Anna, Chan, Curtis M

“…Foust et al . describe a viral vector that crosses the blood-brain barrier, providing a non-invasive method for delivering therapeutic genes to the central…”
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Astrocytes from familial and sporadic ALS patients are toxic to motor neurons by Kaspar, Brian K, Haidet-Phillips, Amanda M, Hester, Mark E, Miranda, Carlos J, Meyer, Kathrin, Braun, Lyndsey, Frakes, Ashley, Song, SungWon, Likhite, Shibi, Murtha, Matthew J, Foust, Kevin D, Rao, Meghan, Eagle, Amy, Kammesheidt, Anja, Christensen, Ashley, Mendell, Jerry R, Burghes, Arthur H M

“…Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease, with astrocytes implicated as contributing substantially to motor neuron death in familial…”
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Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome by Garg, Saurabh K, Lioy, Daniel T, Cheval, Hélène, McGann, James C, Bissonnette, John M, Murtha, Matthew J, Foust, Kevin D, Kaspar, Brian K, Bird, Adrian, Mandel, Gail

“…De novo mutations in the X-linked gene encoding the transcription factor methyl-CpG binding protein 2 (MECP2) are the most frequent cause of the neurological…”
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Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS by Foust, Kevin D, Salazar, Desirée L, Likhite, Shibi, Ferraiuolo, Laura, Ditsworth, Dara, Ilieva, Hristelina, Meyer, Kathrin, Schmelzer, Leah, Braun, Lyndsey, Cleveland, Don W, Kaspar, Brian K

“…Mutations in superoxide dismutase 1 (SOD1) are linked to familial amyotrophic lateral sclerosis (ALS) resulting in progressive motor neuron death through one…”
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Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis by Song, SungWon, Miranda, Carlos J, Braun, Lyndsey, Meyer, Kathrin, Frakes, Ashley E, Ferraiuolo, Laura, Likhite, Shibi, Bevan, Adam K, Foust, Kevin D, McConnell, Michael J, Walker, Christopher M, Kaspar, Brian K

“…Expression of MHC class I molecules on motor neurons protects them from astrocyte-induced toxicity in ALS. Astrocytes isolated from individuals with…”
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A Phase 1/2a Follistatin Gene Therapy Trial for Becker Muscular Dystrophy by 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

“…Becker muscular dystrophy (BMD) is a variant of dystrophin deficiency resulting from DMD gene mutations. Phenotype is variable with loss of ambulation in late…”
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NEUROD1 Instructs Neuronal Conversion in Non-Reactive Astrocytes by Brulet, Rebecca, Matsuda, Taito, Zhang, Ling, Miranda, Carlos, Giacca, Mauro, Kaspar, Brian K., Nakashima, Kinichi, Hsieh, Jenny

“…Currently, all methods for converting non-neuronal cells into neurons involve injury to the brain; however, whether neuronal transdifferentiation can occur…”
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Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates by Samaranch, Lluis, Salegio, Ernesto A, San Sebastian, Waldy, Kells, Adrian P, Foust, Kevin D, Bringas, John R, Lamarre, Clementine, Forsayeth, John, Kaspar, Brian K, Bankiewicz, Krystof S

“…Widespread distribution of gene products at clinically relevant levels throughout the CNS has been challenging. Adeno-associated virus type 9 (AAV9) vector has…”
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Transplantation of cerebellar neural stem cells improves motor coordination and neuropathology in Machado-Joseph disease mice by Mendonça, Liliana S, Nóbrega, Clévio, Hirai, Hirokazu, Kaspar, Brian K, Pereira de Almeida, Luís

“…Machado-Joseph disease is a neurodegenerative disease without effective treatment. Patients with Machado-Joseph disease exhibit significant motor impairments…”
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Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes by 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.

“…Sporadic inclusion body myositis, a variant of inflammatory myopathy, has features distinct from polymyositis/dermatomyositis. The disease affects men more…”
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Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN by Kaspar, Brian K, Foust, Kevin D, Wang, Xueyong, McGovern, Vicki L, Braun, Lyndsey, Bevan, Adam K, Haidet, Amanda M, Le, Thanh T, Morales, Pablo R, Rich, Mark M, Burghes, Arthur H M

“…Spinal muscular atrophy (SMA), the most common autosomal recessive neurodegenerative disease affecting children, results in impaired motor neuron function…”
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