Primary fibroblasts from CSPα mutation carriers recapitulate hallmarks of the adult onset neuronal ceroid lipofuscinosis by Bruno A. Benitez, Mark S. Sands

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Generation of a stable packaging cell line producing high-titer PPT-deleted integration-deficient lentiviral vectors by Peirong Hu, Yedda Li, Mark S Sands, Thomas McCown, Tal Kafri

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CRISPR-Cas9 Knock-In of T513M and G41S Mutations in the Murine β–Galactosyl-Ceramidase Gene Re-capitulates Early-Onset and Adult-Onset Forms of Krabbe Disease by Rima Rebiai, Emily Rue, Steve Zaldua, Duc Nguyen, Giuseppe Scesa, Martin Jastrzebski, Robert Foster, Bin Wang, Xuntian Jiang, Leon Tai, Scott T. Brady, Richard van Breemen, Maria I. Givogri, Mark S. Sands, Mark S. Sands, Ernesto R. Bongarzone

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Compromised astrocyte function and survival negatively impact neurons in infantile neuronal ceroid lipofuscinosis by Jenny Lange, Luke J. Haslett, Emyr Lloyd-Evans, Jennifer M. Pocock, Mark S. Sands, Brenda P. Williams, Jonathan D. Cooper

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Exome-sequencing confirms DNAJC5 mutations as cause of adult neuronal ceroid-lipofuscinosis. by Bruno A Benitez, David Alvarado, Yefei Cai, Kevin Mayo, Sumitra Chakraverty, Joanne Norton, John C Morris, Mark S Sands, Alison Goate, Carlos Cruchaga

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Successive neuron loss in the thalamus and cortex in a mouse model of infantile neuronal ceroid lipofuscinosis by Catherine Kielar, Lucy Maddox, Ellen Bible, Charlie C. Pontikis, Shannon L. Macauley, Megan A. Griffey, Michael Wong, Mark S. Sands, Jonathan D. Cooper

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Effects of chronic cannabidiol in a mouse model of naturally occurring neuroinflammation, neurodegeneration, and spontaneous seizures by Joshua T. Dearborn, Hemanth R. Nelvagal, Nicholas R. Rensing, Keigo Takahashi, Stephanie M. Hughes, Thomas M. Wishart, Jonathan D. Cooper, Michael Wong, Mark S. Sands

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Development of sensory, motor and behavioral deficits in the murine model of Sanfilippo syndrome type B. by Coy D Heldermon, Anne K Hennig, Kevin K Ohlemiller, Judith M Ogilvie, Erik D Herzog, Annalisa Breidenbach, Carole Vogler, David F Wozniak, Mark S Sands

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Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model by Keigo Takahashi, Elizabeth M. Eultgen, Sophie H. Wang, Nicholas R. Rensing, Hemanth R. Nelvagal, Joshua T. Dearborn, Olivier Danos, Nicholas Buss, Mark S. Sands, Michael Wong, Jonathan D. Cooper

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A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I by Steven Q. Le, Shih-hsin Kan, Don Clarke, Valentina Sanghez, Martin Egeland, Kristen N. Vondrak, Terence M. Doherty, Moin U. Vera, Michelina Iacovino, Jonathan D. Cooper, Mark S. Sands, Patricia I. Dickson

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Embryonic vitamin D deficiency programs hematopoietic stem cells to induce type 2 diabetes by Jisu Oh, Amy E. Riek, Kevin T. Bauerle, Adriana Dusso, Kyle P. McNerney, Ruteja A. Barve, Isra Darwech, Jennifer E. Sprague, Clare Moynihan, Rong M. Zhang, Greta Kutz, Ting Wang, Xiaoyun Xing, Daofeng Li, Marguerite Mrad, Nicholas M. Wigge, Esmeralda Castelblanco, Alejandro Collin, Monika Bambouskova, Richard D. Head, Mark S. Sands, Carlos Bernal-Mizrachi

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

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