Results 121 to 130 of about 65,862 (234)

Ready-to-use iPSC-derived microglia progenitors for the treatment of CNS disease in mouse models of neuropathic mucopolysaccharidoses

Nature Communications
Mucopolysaccharidoses are inherited metabolic disorders caused by the deficiency in lysosomal enzymes required to break down glycosaminoglycans. Accumulation of glycosaminoglycans leads to progressive, systemic degenerative disease.
Panagiotis Douvaras, Diego F. Buenaventura, Bruce Sun, Ashley Lepack, Elizabeth Baker, Elizabeth Simpson, Mark Ebel, Gregory Lallos, Deven LoSchiavo, Nicholas Stitt, Nathaniel Adams, Conor McAuliffe, Ana Forton-Juarez, Brian Kosmyna, Elizabeth Pereira, Benjamin Burnett, David Dilworth, Stephanie Fisher, Jing Wang, Peter Tonge, Mark Tomishima, Carlos Paladini, Dan Wilkinson, Chew-Li Soh, Maya Srinivas, Christoph Patsch, Stefan Irion   +26 more
doaj   +1 more source

Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease

Frontiers in Neurology
Enzyme replacement therapy (ERT) is the only approved disease-modifying treatment modality for Pompe disease, a rare, inherited metabolic disorder caused by a deficiency in the acid α-glucosidase (GAA) enzyme that catabolizes lysosomal glycogen.
Barry J. Byrne, Giancarlo Parenti, Giancarlo Parenti, Benedikt Schoser, Ans T. van der Ploeg, Hung Do, Brian Fox, Mitchell Goldman, Franklin K. Johnson, Jia Kang, Nickita Mehta, John Mondick, M. Osman Sheikh, Sheela Sitaraman Das, Steven Tuske, Jon Brudvig, Jill M. Weimer, Tahseen Mozaffar   +17 more
doaj   +1 more source

Biochemical evidence that the type II insulin-like growth factor receptor is identical to the cation-independent mannose 6-phosphate receptor.

, 1988
Wieland Kieß, G D Blickenstaff, Mark Sklar, Cheryl L. Thomas, S. Peter Nissley, G G Sahagian   +5 more
openalex   +1 more source

Exocytosis of Progeny Infectious Varicella-Zoster Virus Particles via a Mannose-6-Phosphate Receptor Pathway without Xenophagy following Secondary Envelopment. [PDF]

J Virol, 2020
Girsch JH, Jackson W, Carpenter JE, Moninger TO, Jarosinski KW, Grose C.   +5 more
europepmc   +1 more source

Conversion of G-protein specificity of insulin-like growth factor II/mannose 6-phosphate receptor by exchanging of a short region with beta-adrenergic receptor. [PDF]

, 1993
Kyôko Takahashi, Yoshitake Murayama, Takashi Okamoto, Takashi Yokota, Tsuneya Ikezu, S Takahashi, Ugo Giambarella, Etsuro Ogata, Ikuo Nishimoto   +8 more
openalex   +1 more source

VPS35 dysfunction impairs lysosomal degradation of α-synuclein and exacerbates neurotoxicity in a Drosophila model of Parkinson's disease

Neurobiology of Disease, 2014
Mutations in vacuolar protein sorting 35 (VPS35) have been linked to familial Parkinson's disease (PD). VPS35, a component of the retromer, mediates the retrograde transport of cargo from the endosome to the trans-Golgi network.
Emiko Miura, Takafumi Hasegawa, Masatoshi Konno, Mari Suzuki, Naoto Sugeno, Nobuhiro Fujikake, Sven Geisler, Mitsuaki Tabuchi, Ryuji Oshima, Akio Kikuchi, Toru Baba, Keiji Wada, Yoshitaka Nagai, Atsushi Takeda, Masashi Aoki   +14 more
doaj  

Serum Form of the Rat Insulin-like Growth Factor II/Mannose 6-Phosphate Receptor Is Truncated in the Carboxyl-terminal Domain

, 1989
Richard G. MacDonald, Mark A. Tepper, Kevin B. Clairmont, Susan B. Perregaux, M P Czech   +4 more
openalex   +1 more source

Mannose 6–Phosphate Receptors Are Sorted from Immature Secretory Granules via Adaptor Protein AP-1, Clathrin, and Syntaxin 6–positive Vesicles [PDF]

, 1998
Judith Klumperman, Regina Kuliawat, Janice Griffith, Hans J. Geuze, Peter Arvan   +4 more
openalex   +1 more source

Neither type of mannose 6-phosphate receptor is sufficient for targeting of lysosomal enzymes along intracellular routes. [PDF]

, 1996
Dagmar Kasper, Frank Dittmer, Kurt Von Figura, Regina Pohlmann   +3 more
openalex   +1 more source

Recognition and receptor-mediated endocytosis of the lysosomal acid lipase secreted by cultured human fibroblasts.

Journal of Lipid Research, 1982
We have studied the recognition and uptake of acid lipase by human fibroblasts in order to determine requirements for localization and function of the enzyme in lysosomes.
G N Sando, V L Henke
doaj