Ragulator-a multifaceted regulator of lysosomal signaling and trafficking von Colaço, Alexandria, Jäättelä, Marja

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STAT3 associates with vacuolar H+-ATPase and regulates cytosolic and lysosomal pH von Liu, Bin, Palmfeldt, Johan, Lin, Lin, Colaço, Alexandria, Clemmensen, Knut K. B., Huang, Jinrong, Xu, Fengping, Liu, Xin, Maeda, Kenji, Luo, Yonglun, Jäättelä, Marja

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FTY720/fingolimod increases NPC1 and NPC2 expression and reduces cholesterol and sphingolipid accumulation in Niemann‐Pick type C mutant fibroblasts von Newton, Jason, Hait, Nitai C., Maceyka, Michael, Colaco, Alexandria, Maczis, Melissa, Wassif, Christopher A., Cougnoux, Antony, Porter, Forbes D., Milstien, Sheldon, Platt, Nicholas, Platt, Frances M., Spiegel, Sarah

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A comparative study on fluorescent cholesterol analogs as versatile cellular reporters[S] von Sezgin, Erdinc, Can, Fatma Betul, Schneider, Falk, Clausen, Mathias P., Galiani, Silvia, Stanly, Tess A., Waithe, Dominic, Colaco, Alexandria, Honigmann, Alf, Wüstner, Daniel, Platt, Frances, Eggeling, Christian

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Immune dysfunction in Niemann‐Pick disease type C von Platt, Nick, Speak, Annelise O., Colaco, Alexandria, Gray, James, Smith, David A., Williams, Ian M., Wallom, Kerri‐Lee, Platt, Frances M.

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Mechanistic convergence and shared therapeutic targets in Niemann‐Pick disease von Colaco, Alexandria, Kaya, Ecem, Adriaenssens, Elias, Davis, Lianne C., Zampieri, Stefania, Fernández‐Suárez, María E., Tan, Chong Y., Deegan, Patrick B., Porter, Forbes D., Galione, Antony, Bembi, Bruno, Dardis, Andrea, Platt, Frances M.

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Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease von Bradbury, Allison M., Gray-Edwards, Heather L., Shirley, Jamie L., McCurdy, Victoria J., Colaco, Alexandria N., Randle, Ashley N., Christopherson, Pete W., Bird, Allison C., Johnson, Aime K., Wilson, Diane U., Hudson, Judith A., De Pompa, Nicholas L., Sorjonen, Donald C., Brunson, Brandon L., Jeyakumar, Mylvaganam, Platt, Frances M., Baker, Henry J., Cox, Nancy R., Sena-Esteves, Miguel, Martin, Douglas R.

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Defective iron homeostasis and hematological abnormalities in Niemann-Pick disease type C1 von Chen, Oscar C W, Siebel, Stephan, Colaco, Alexandria, Nicoli, Elena-Raluca, Platt, Nick, Shepherd, Dawn, Newman, Stephanie, Armitage, Andrew E, Farhat, Nicole Y, Seligmann, George, Smith, Claire, Smith, David A, Abdul-Sada, Alaa, Jeyakumar, Mylvaganam, Drakesmith, Hal, Porter, Forbes D, Platt, Frances M

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Defective platelet function in Niemann‐Pick disease type C1 von Chen, Oscar C. W., Colaco, Alexandria, Davis, Lianne C., Kiskin, Fedir N., Farhat, Nicole Y., Speak, Anneliese O., Smith, David A., Morris, Lauren, Eden, Emily, Tynan, Patricia, Churchill, Grant C., Galione, Antony, Porter, Forbes D., Platt, Frances M.

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Drug‐induced increase in lysobisphosphatidic acid reduces the cholesterol overload in Niemann–Pick type C cells and mice von Moreau, Dimitri, Vacca, Fabrizio, Vossio, Stefania, Scott, Cameron, Colaco, Alexandria, Paz Montoya, Jonathan, Ferguson, Charles, Damme, Markus, Moniatte, Marc, Parton, Robert G, Platt, Frances M, Gruenberg, Jean

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AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease von Hughes, Michael P, Smith, Dave A, Morris, Lauren, Fletcher, Claire, Colaco, Alexandria, Huebecker, Mylene, Tordo, Julie, Palomar, Nuria, Massaro, Giulia, Henckaerts, Els, Waddington, Simon N, Platt, Frances M, Rahim, Ahad A

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Drug-induced increase in lysobisphosphatidic acid reduces the cholesterol overload in Niemann-Pick type C cells and mice von Moreau, Dimitri, Vacca, Fabrizio, Vossio, Stefania, Scott, Cameron, Colaco, Alexandria, Paz Montoya, Jonathan, Ferguson, Charles, Damme, Markus, Moniatte, Marc, Parton, Robert G, Platt, Frances M, Gruenberg, Jean

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Disorders of Cholesterol Metabolism and Their Unanticipated Convergent Mechanisms of Disease von Platt, Frances M, Wassif, Christopher, Colaco, Alexandria, Dardis, Andrea, Lloyd-Evans, Emyr, Bembi, Bruno, Porter, Forbes D

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AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease von Hughes, Michael P, Smith, Dave A, Morris, Lauren, Fletcher, Claire, Colaco, Alexandria, Huebecker, Mylene, Tordo, Julie, Palomar, Nuria, Massaro, Giulia, Henckaerts, Els, Waddington, Simon N, Platt, Frances M, Rahim, Ahad A

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Defective iron homeostasis and hematological abnormalities in Niemann-Pick disease type C1 von Chen, Oscar C W, Siebel, Stephan, Colaco, Alexandria, Nicoli, Elena-Raluca, Platt, Nick, Shepherd, Dawn, Newman, Stephanie, Armitage, Andrew E, Farhat, Nicole Y, Seligmann, George, Smith, Claire, Smith, David A, Abdul-Sada, Alaa, Jeyakumar, Mylvaganam, Drakesmith, Hal, Porter, Forbes D, Platt, Frances M

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Unbiased yeast screens identify cellular pathways affected in Niemann-Pick disease type C von Colaco, Alexandria, Fernández-Suárez, María E, Shepherd, Dawn, Gal, Lihi, Bibi, Chen, Chuartzman, Silvia, Diot, Alan, Morten, Karl, Eden, Emily, Porter, Forbes D, Poulton, Joanna, Platt, Nick, Schuldiner, Maya, Platt, Frances M

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Drug-induced increase in lysobisphosphatidic acid reduces the cholesterol overload in Niemann-Pick type C cells and mice von Moreau, Dimitri, Vacca, Fabrizio, Vossio, Stefania, Scott, Cameron, Colaco, Alexandria, Montoya, Jonathan Paz, Ferguson, Charles, Damme, Markus, Moniatte, Marc, Parton, Robert G, Platt, Frances M, Gruenberg, Jean

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Development, application and evaluation of a computational tool for management high voltage break disconnector based on self-organizing maps and image processing von Colaço, Daniel Freitas, de Alexandria, Auzuir R., Cortez, Paulo César, Frota, João Batista B., Lima, José Nunes de, de Albuquerque, Victor Hugo C.

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