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Suppression of autophagy permits successful enzyme replacement therapy in a lysosomal storage disorder-murine Pompe disease
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Published in Autophagy (16.11.2010)
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Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease
Fukuda, Tokiko, Ewan, Lindsay, Bauer, Martina, Mattaliano, Robert J., Zaal, Kristien, Ralston, Evelyn, Plotz, Paul H., Raben, Nina
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Rider, Lisa G., Koziol, Deloris, Giannini, Edward H., Jain, Minal S., Smith, Michaele R., Whitney‐Mahoney, Kristi, Feldman, Brian M., Wright, Susan J., Lindsley, Carol B., Pachman, Lauren M., Villalba, Maria L., Lovell, Daniel J., Bowyer, Suzanne L., Plotz, Paul H., Miller, Frederick W., Hicks, Jeanne E.
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Autophagy and Mistargeting of Therapeutic Enzyme in Skeletal Muscle in Pompe Disease
Fukuda, Tokiko, Ahearn, Meghan, Roberts, Ashley, Mattaliano, Robert J, Zaal, Kristien, Ralston, Evelyn, Plotz, Paul H, Raben, Nina
Published in Molecular therapy (01.12.2006)
Published in Molecular therapy (01.12.2006)
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Autophagy and Lysosomes in Pompe Disease
FUKUDA, TOKIKO, Roberts, Ashley, Ahearn, Meghan, Zaal, Kristien, Ralston, Evelyn, Plotz, Paul H., Raben, Nina
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Published in Autophagy (01.10.2006)
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Deconstructing Pompe Disease by Analyzing Single Muscle Fibers: "To See a World in a Grain of Sand..."
Raben, Nina, Takikita, Shoichi, Pittis, Maria G., Bembi, Bruno, Marie, Suely K.N., Roberts, Ashley, Page, Laura, Kishnani, Priya S., Schoser, Benedikt G.H., Chien, Yin-Hsiu, Ralston, Evelyn, Nagaraju, Kanneboyina, Plotz, Paul H.
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Published in Autophagy (26.11.2007)
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Correction of the Enzymatic and Functional Deficits in a Model of Pompe Disease Using Adeno-associated Virus Vectors
Fraites, Thomas J., Schleissing, Mary R., Shanely, R.Andrew, Walter, Glenn A., Cloutier, Denise A., Zolotukhin, Irene, Pauly, Daniel F., Raben, Nina, Plotz, Paul H., Powers, Scott K., Kessler, Paul D., Byrne, Barry J.
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Published in Molecular therapy (01.05.2002)
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Published in The Lancet (British edition) (22.11.2003)
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Moutsopoulos, Haralampos M, Talal, Marilynn, Plotz, Paul H
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Controlled Trial of Plasma Exchange and Leukapheresis in Polymyositis and Dermatomyositis
Miller, Frederick W, Leitman, Susan F, Cronin, Mary E, Hicks, Jeanne E, Leff, Richard L, Wesley, Robert, Fraser, David D, Dalakas, Marinos, Plotz, Paul H
Published in The New England journal of medicine (21.05.1992)
Published in The New England journal of medicine (21.05.1992)
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Differences in the predominance of lysosomal and autophagic pathologies between infants and adults with Pompe disease: implications for therapy
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Published in Molecular genetics and metabolism (01.12.2010)
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Published in The Journal of experimental medicine (16.09.2002)
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