Mucopolysaccharidosis and Autophagy: Controversies on the Contribution of the Process to the Pathogenesis and Possible Therapeutic Applications.

Neuromolecular Medicine
Karolina PierzynowskaGrzegorz Węgrzyn

Abstract

Mucopolysaccharidosis (MPS) consists of a group of 11 enzymatic defects which result in accumulation of undegraded glycosaminoglycans (GAG) in lysosomes. MPS is a severe metabolic disease for which only bone marrow/hematopoietic stem cell transplantation and enzyme replacement therapy are current therapeutic options. However, they are available for only a few of MPS types, and are ineffective in treatment of central nervous system. Recent studies indicated that the autophagy process can be impaired in MPS, but various contradictory conclusions have been published in this matter. Nevertheless, stimulation of autophagy has been proposed as a potential therapeutic option for MPS, and very recent results suggest that such approach might be effective in improving MPS symptoms. Still the mechanisms of autophagy changes in MPS are not clear, and efficiency of autophagy activation in clearing the storage material requires further investigation. These problems are summarized and discussed in this review.

References

Jan 17, 2007·Autophagy·Alfred J Meijer, Patrice Codogno
Oct 5, 2007·Human Molecular Genetics·Carmine SettembreAndrea Ballabio
Nov 15, 2007·Autophagy·Carmine SettembreAndrea Ballabio
Aug 25, 2009·The Journal of Biological Chemistry·Josh C WoloszynekMark S Sands
Nov 3, 2010·The American Journal of Pathology·Sandrine VitryJean Michel Heard
Feb 2, 2011·Journal of Cell Science·Susmita KaushikAna Maria Cuervo
Jun 8, 2011·Autophagy·Dalibor MijaljicaRodney J Devenish
Apr 29, 2014·The Journal of Biological Chemistry·Marta MoskotMagdalena Gabig-Cimińska
Sep 1, 2015·Experimental Eye Research·Roy KarnatiGordon W Laurie
Aug 1, 2004·Molecular and Cellular Biochemistry·Ana Maria Cuervo
Feb 22, 2017·JIMD Reports·Gustavo Monteiro VianaVânia D'Almeida
Sep 6, 2017·The Journal of Clinical Investigation·Rosa BartolomeoCarmine Settembre
Feb 13, 2018·Neuromolecular Medicine·Karolina PierzynowskaGrzegorz Węgrzyn
Mar 16, 2018·Metabolic Brain Disease·Karolina PierzynowskaGrzegorz Węgrzyn
May 23, 2018·Biomedicine & Pharmacotherapy = Biomédecine & Pharmacothérapie·Jelena JakovljevicVladislav Volarevic
Aug 18, 2018·Nature Reviews. Drug Discovery·Barry BolandMark J Millan
Sep 6, 2018·Cancer Chemotherapy and Pharmacology·Eleanor Bishop, Tracey D Bradshaw
Sep 17, 2018·Journal of Autoimmunity·Srinivasa Reddy BonamSylviane Muller
Sep 19, 2018·Cancer Chemotherapy and Pharmacology·Lei ZhanBing Wei

❮ Previous
Next ❯

Citations

Sep 19, 2020·Journal of Inherited Metabolic Disease·Berna Seker YilmazJulien Baruteau
Mar 29, 2020·Frontiers in Molecular Neuroscience·Antonio Monaco, Alessandro Fraldi
Aug 17, 2020·Journal of Clinical Medicine·Karolina M StepienIain Hargreaves
Nov 17, 2020·Frontiers in Cell and Developmental Biology·Karolina PierzynowskaGrzegorz Węgrzyn
Jan 1, 2021·International Journal of Molecular Sciences·Víctor J ÁlvarezMaría L Couce
Mar 4, 2021·Expert Opinion on Biological Therapy·Azam SafaryYadollah Omidi
Oct 17, 2020·Current Osteoporosis Reports·Zhirui JiangLachlan J Smith
Sep 3, 2020·European Journal of Pharmacology·Estera RintzGrzegorz Węgrzyn

❮ Previous
Next ❯

Related Concepts

Related Feeds

Autophagy & Model Organisms

Autophagy is a cellular process that allows degradation by the lysosome of cytoplasmic components such as proteins or organelles. Here is the latest research on autophagy & model organisms

Related Papers

American Journal of Medical Genetics. Part a
Taiane VieiraRoberto Giugliani
British Medical Journal
L Pavitt
AJNR. American Journal of Neuroradiology
R MurataY Inoue
Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics
Yong-Lan HuangLi Liu
© 2021 Meta ULC. All rights reserved