Autophagy: the pathogenic agent in muscle damage

por Raben, Nina
Publicado 2013

Pompe Disease: New Developments in an Old Lysosomal Storage Disorder

por Meena, Naresh K., Raben, Nina
Publicado 2020

Impaired autophagy: The collateral damage of lysosomal storage disorders

por Myerowitz, Rachel, Puertollano, Rosa, Raben, Nina
Publicado 2020

Pompe disease: from pathophysiology to therapy and back again

por Lim, Jeong-A, Li, Lishu, Raben, Nina
Publicado 2014

AAV-mediated delivery of secreted acid α-glucosidase with enhanced uptake corrects neuromuscular pathology in Pompe mice

por Meena, Naresh K., Randazzo, Davide, Raben, Nina, Puertollano, Rosa
Publicado 2023

Atg5(flox)-Derived Autophagy-Deficient Model of Pompe Disease: Does It Tell the Whole Story?

por Lim, Jeong-A, Zare, Hossein, Puertollano, Rosa, Raben, Nina
Publicado 2017

Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease

por Meena, Naresh Kumar, Ralston, Evelyn, Raben, Nina, Puertollano, Rosa
Publicado 2020

Pompe disease: Shared and unshared features of lysosomal storage disorders

por Lim, Jeong-A, Kakhlon, Or, Li, Lishu, Myerowitz, Rachel, Raben, Nina
Publicado 2015

Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease

por Lim, Jeong-A, Yi, Haiqing, Gao, Fengqin, Raben, Nina, Kishnani, Priya S., Sun, Baodong
Publicado 2019

Autophagy in lysosomal storage disorders

por Lieberman, Andrew P., Puertollano, Rosa, Raben, Nina, Slaugenhaupt, Susan, Walkley, Steven U., Ballabio, Andrea
Publicado 2012

Modulation of mTOR signaling as a strategy for the treatment of Pompe disease

por Lim, Jeong‐A, Li, Lishu, Shirihai, Orian S, Trudeau, Kyle M, Puertollano, Rosa, Raben, Nina
Publicado 2017

Fiber Type Conversion by PGC-1α Activates Lysosomal and Autophagosomal Biogenesis in Both Unaffected and Pompe Skeletal Muscle

por Takikita, Shoichi, Schreiner, Cynthia, Baum, Rebecca, Xie, Tao, Ralston, Evelyn, Plotz, Paul H., Raben, Nina
Publicado 2010

Chemoenzymatic glycan-selective remodeling of a therapeutic lysosomal enzyme with high-affinity M6P-glycan ligands. Enzyme substrate specificity is the name of the game

por Zhang, Xiao, Liu, Huiying, Meena, Naresh, Li, Chao, Zong, Guanghui, Raben, Nina, Puertollano, Rosa, Wang, Lai-Xi
Publicado 2021

Skeletal muscle pathology of infantile Pompe disease during long-term enzyme replacement therapy

por Prater, Sean N, Patel, Trusha T, Buckley, Anne F, Mandel, Hanna, Vlodavski, Eugene, Banugaria, Suhrad G, Feeney, Erin J, Raben, Nina, Kishnani, Priya S
Publicado 2013

The value of muscle biopsies in Pompe disease: identifying lipofuscin inclusions in juvenile- and adult-onset patients

por Feeney, Erin J, Austin, Stephanie, Chien, Yin-Hsiu, Mandel, Hanna, Schoser, Benedikt, Prater, Sean, Hwu, Wuh-Liang, Ralston, Evelyn, Kishnani, Priya S, Raben, Nina
Publicado 2014

Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease

por Spampanato, Carmine, Feeney, Erin, Li, Lishu, Cardone, Monica, Lim, Jeong-A, Annunziata, Fabio, Zare, Hossein, Polishchuk, Roman, Puertollano, Rosa, Parenti, Giancarlo, Ballabio, Andrea, Raben, Nina
Publicado 2013

Histidyl–tRNA Synthetase and Asparaginyl–tRNA Synthetase, Autoantigens in Myositis, Activate Chemokine Receptors on T Lymphocytes and Immature Dendritic Cells

por Howard, O.M. Zack, Dong, Hui Fang, Yang, De, Raben, Nina, Nagaraju, Kanneboyina, Rosen, Antony, Casciola-Rosen, Livia, Härtlein, Michael, Kron, Michael, Yang, David, Yiadom, Kwabena, Dwivedi, Sunita, Plotz, Paul H., Oppenheim, Joost J.
Publicado 2002