Search Results - "Nelvagal, Hemanth R"

Pathomechanisms in the neuronal ceroid lipofuscinoses by Nelvagal, Hemanth R., Lange, Jenny, Takahashi, Keigo, Tarczyluk-Wells, Marta A., Cooper, Jonathan D.

“…The neuronal ceroid lipofuscinoses (NCLs) are a group of inherited neurodegenerative lysosomal storage disorders (LSDs), traditionally grouped together based…”
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mTORC1-independent TFEB activation via Akt inhibition promotes cellular clearance in neurodegenerative storage diseases by Palmieri, Michela, Pal, Rituraj, Nelvagal, Hemanth R., Lotfi, Parisa, Stinnett, Gary R., Seymour, Michelle L., Chaudhury, Arindam, Bajaj, Lakshya, Bondar, Vitaliy V., Bremner, Laura, Saleem, Usama, Tse, Dennis Y., Sanagasetti, Deepthi, Wu, Samuel M., Neilson, Joel R., Pereira, Fred A., Pautler, Robia G., Rodney, George G., Cooper, Jonathan D., Sardiello, Marco

“…Neurodegenerative diseases characterized by aberrant accumulation of undigested cellular components represent unmet medical conditions for which the…”
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Synergistic effects of treating the spinal cord and brain in CLN1 disease by Shyng, Charles, Nelvagal, Hemanth R., Dearborn, Joshua T., Tyynelä, Jaana, Schmidt, Robert E., Sands, Mark S., Cooper, Jonathan D.

“…Infantile neuronal ceroid lipofuscinosis (INCL, or CLN1 disease) is an inherited neurodegenerative storage disorder caused by a deficiency of the lysosomal…”
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Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model by Takahashi, Keigo, Eultgen, Elizabeth M, Wang, Sophie H, Rensing, Nicholas R, Nelvagal, Hemanth R, Dearborn, Joshua T, Danos, Olivier, Buss, Nicholas, Sands, Mark S, Wong, Michael, Cooper, Jonathan D

“…Although a disease-modifying therapy for classic late infantile neuronal ceroid lipofuscinosis (CLN2 disease) exists, poor understanding of cellular…”
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Top-down and bottom-up propagation of disease in the neuronal ceroid lipofuscinoses by Ostergaard, John R., Nelvagal, Hemanth R., Cooper, Jonathan D.

“…Background The Neuronal Ceroid Lipofuscinoses (NCLs) may be considered distinct neurodegenerative disorders with separate underlying molecular causes resulting…”
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Glial Dysfunction and Its Contribution to the Pathogenesis of the Neuronal Ceroid Lipofuscinoses by Takahashi, Keigo, Nelvagal, Hemanth R, Lange, Jenny, Cooper, Jonathan D

“…While significant efforts have been made in developing pre-clinical treatments for the neuronal ceroid lipofuscinoses (NCLs), many challenges still remain to…”
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Effects of chronic cannabidiol in a mouse model of naturally occurring neuroinflammation, neurodegeneration, and spontaneous seizures by Dearborn, Joshua T., Nelvagal, Hemanth R., Rensing, Nicholas R., Takahashi, Keigo, Hughes, Stephanie M., Wishart, Thomas M., Cooper, Jonathan D., Wong, Michael, Sands, Mark S.

“…Cannabidiol (CBD) has gained attention as a therapeutic agent and is purported to have immunomodulatory, neuroprotective, and anti-seizure effects. Here, we…”
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Substrate reduction therapy for Krabbe disease and metachromatic leukodystrophy using a novel ceramide galactosyltransferase inhibitor by Babcock, Michael C., Mikulka, Christina R., Wang, Bing, Chandriani, Sanjay, Chandra, Sundeep, Xu, Yue, Webster, Katherine, Feng, Ying, Nelvagal, Hemanth R., Giaramita, Alex, Yip, Bryan K., Lo, Melanie, Jiang, Xuntian, Chao, Qi, Woloszynek, Josh C., Shen, Yuqiao, Bhagwat, Shripad, Sands, Mark S., Crawford, Brett E.

“…Krabbe disease (KD) and metachromatic leukodystrophy (MLD) are caused by accumulation of the glycolipids galactosylceramide (GalCer) and sulfatide and their…”
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Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep by Nelvagal, Hemanth R, Eaton, Samantha L, Wang, Sophie H, Eultgen, Elizabeth M, Takahashi, Keigo, Le, Steven Q, Nesbitt, Rachel, Dearborn, Joshua T, Siano, Nicholas, Puhl, Ana C, Dickson, Patricia I, Thompson, Gerard, Murdoch, Fraser, Brennan, Paul M, Gray, Mark, Greenhalgh, Stephen N, Tennant, Peter, Gregson, Rachael, Clutton, Eddie, Nixon, James, Proudfoot, Chris, Guido, Stefano, Lillico, Simon G, Whitelaw, C Bruce A, Lu, Jui-Yun, Hofmann, Sandra L, Ekins, Sean, Sands, Mark S, Wishart, Thomas M, Cooper, Jonathan D

“…CLN1 disease, also called infantile neuronal ceroid lipofuscinosis (NCL) or infantile Batten disease, is a fatal neurodegenerative lysosomal storage disorder…”
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Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis by Lu, Jui-Yun, Nelvagal, Hemanth R., Wang, Lingling, Birnbaum, Shari G., Cooper, Jonathan D., Hofmann, Sandra L.

“…The neuronal ceroid lipofuscinoses (NCLs) are a group of related hereditary lysosomal storage disorders characterized by progressive loss of neurons in the…”
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Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice by White, Katherine A., Nelvagal, Hemanth R., Poole, Timothy A., Lu, Bin, Johnson, Tyler B., Davis, Samantha, Pratt, Melissa A., Brudvig, Jon, Assis, Ana B., Likhite, Shibi, Meyer, Kathrin, Kaspar, Brian K., Cooper, Jonathan D., Wang, Shaomei, Weimer, Jill M.

“…Batten disease is a family of rare, fatal, neuropediatric diseases presenting with memory/learning decline, blindness, and loss of motor function. Recently, we…”
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Towards a new understanding of NCL pathogenesis by Cooper, Jonathan D., Tarczyluk, Marta A., Nelvagal, Hemanth R.

“…The Neuronal Ceroid Lipofuscinoses (NCLs, Batten disease) are a group of inherited neurodegenerative disorders that have been traditionally grouped together on…”
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Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease by Massaro, Giulia, Geard, Amy F, Nelvagal, Hemanth R, Gore, Katrina, Clemo, Nadine K, Waddington, Simon N, Rahim, Ahad A

“…Abstract Gaucher Disease (GD) is an inherited metabolic disorder caused by mutations in the GBA1 gene. It can manifest with severe neurodegeneration and…”
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Progress toward Fulfilling the Potential of Immunomodulation in Childhood Neurodegeneration? by Cooper, Jonathan D., Nelvagal, Hemanth R.

“…[...]a recent pre-clinical study has provided evidence that systemic delivery of a self-complementary AAV9 vector partly corrects pathology in Cln3Δex7/8…”
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Comparative proteomic profiling reveals mechanisms for early spinal cord vulnerability in CLN1 disease by Nelvagal, Hemanth R., Hurtado, Maica Llavero, Eaton, Samantha L., Kline, Rachel A., Lamont, Douglas J., Sands, Mark S., Wishart, Thomas M., Cooper, Jonathan D.

“…CLN1 disease is a fatal inherited neurodegenerative lysosomal storage disease of early childhood, caused by mutations in the CLN1 gene, which encodes the…”
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Immune cells perturb axons and impair neuronal survival in a mouse model of infantile neuronal ceroid lipofuscinosis by Groh, Janos, Kühl, Thomas G, Ip, Chi Wang, Nelvagal, Hemanth R, Sri, Sarmi, Duckett, Steven, Mirza, Myriam, Langmann, Thomas, Cooper, Jonathan D, Martini, Rudolf

“…The neuronal ceroid lipofuscinoses are fatal neurodegenerative disorders in which the visual system is affected early in disease progression. A typical…”
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Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB by Ellinwood, N. Matthew, Valentine, Bethann N., Hess, Andrew S., Jens, Jackie K., Snella, Elizabeth M., Jamil, Maryam, Hostetter, Shannon J., Jeffery, Nicholas D., Smith, Jodi D., Millman, Suzanne T., Parsons, Rebecca L., Butt, Mark T., Chandra, Sundeep, Egeland, Martin T., Assis, Ana B., Nelvagal, Hemanth R., Cooper, Jonathan D., Nestrasil, Igor, Mueller, Bryon A., Labounek, Rene, Paulson, Amy, Prill, Heather, Liu, Xiao Ying, Zhou, Huiyu, Lawrence, Roger, Crawford, Brett E., Grover, Anita, Cherala, Ganesh, Melton, Andrew C., Cherukuri, Anu, Vuillemenot, Brian R., Wait, Jill C.M., O’Neill, Charles A., Pinkstaff, Jason, Kovalchin, Joseph, Zanelli, Eric, McCullagh, Emma

“…Mucopolysaccharidosis type IIIB (MPS IIIB; Sanfilippo syndrome B; OMIM #252920) is a lethal, pediatric, neuropathic, autosomal recessive, and lysosomal storage…”
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Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene by Sondhi, Dolan, Scott, Emma C, Chen, Alvin, Hackett, Neil R, Wong, Andrew M S, Kubiak, Agnieszka, Nelvagal, Hemanth R, Pearse, Yewande, Cotman, Susan L, Cooper, Jonathan D, Crystal, Ronald G

“…Juvenile neuronal ceroid lipofuscinosis (JNCL or CLN3 disease) is an autosomal recessive lysosomal storage disease resulting from mutations in the CLN3 gene…”
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Neuronal deletion of the circadian clock gene Bmal1 induces cell-autonomous dopaminergic neurodegeneration by Kanan, Michael F, Sheehan, Patrick W, Haines, Jessica N, Gomez, Pedro G, Dhuler, Adya, Nadarajah, Collin J, Wargel, Zachary M, Freeberg, Brittany M, Nelvagal, Hemanth R, Izumo, Mariko, Takahashi, Joseph S, Cooper, Jonathan D, Davis, Albert A, Musiek, Erik S

“…Circadian rhythm dysfunction is a hallmark of Parkinson disease (PD), and diminished expression of the core clock gene Bmal1 has been described in patients…”
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Corrigendum: mTORC1-independent TFEB activation via Akt inhibition promotes cellular clearance in neurodegenerative storage diseases by Palmieri, Michela, Pal, Rituraj, Nelvagal, Hemanth R, Lotfi, Parisa, Stinnett, Gary R, Seymour, Michelle L, Chaudhury, Arindam, Bajaj, Lakshya, Bondar, Vitaliy V, Bremner, Laura, Saleem, Usama, Tse, Dennis Y, Sanagasetti, Deepthi, Wu, Samuel M, Neilson, Joel R, Pereira, Fred A, Pautler, Robia G, Rodney, George G, Cooper, Jonathan D, Sardiello, Marco

“…This corrects the article DOI: 10.1038/ncomms14338…”
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