Pros and cons of different ways to address dysfunctional autophagy in Pompe disease
Identifieur interne : 000513 ( Main/Exploration ); précédent : 000512; suivant : 000514Pros and cons of different ways to address dysfunctional autophagy in Pompe disease
Auteurs : Jeong-A Lim [États-Unis] ; Naresh Kumar Meena [États-Unis] ; Nina Raben [États-Unis]Source :
- Annals of Translational Medicine [ 2305-5839 ] ; 2019.
Abstract
Autophagy is a major intracellular self-digestion process that brings cytoplasmic materials to the lysosome for degradation. Defective autophagy has been linked to a broad range of human disorders, including cancer, diabetes, neurodegeneration, autoimmunity, cardiovascular diseases, and myopathies. In Pompe disease, a severe neuromuscular disorder, disturbances in autophagic process manifest themselves as progressive accumulation of undegraded cellular debris in the diseased muscle cells. A growing body of evidence has connected this defect to the decline in muscle function and muscle resistance to the currently available treatment—enzyme replacement therapy (ERT). Both induction and inhibition of autophagy have been tested in pre-clinical studies in a mouse model of the disease. Here, we discuss strengths and weaknesses of different approaches to address autophagic dysfunction in the context of Pompe disease.
Url:
DOI: 10.21037/atm.2019.03.51
PubMed: 31392191
PubMed Central: 6642936
Affiliations:
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Defective autophagy has been linked to a broad range of human disorders, including cancer, diabetes, neurodegeneration, autoimmunity, cardiovascular diseases, and myopathies. In Pompe disease, a severe neuromuscular disorder, disturbances in autophagic process manifest themselves as progressive accumulation of undegraded cellular debris in the diseased muscle cells. A growing body of evidence has connected this defect to the decline in muscle function and muscle resistance to the currently available treatment—enzyme replacement therapy (ERT). Both induction and inhibition of autophagy have been tested in pre-clinical studies in a mouse model of the disease. Here, we discuss strengths and weaknesses of different approaches to address autophagic dysfunction in the context of Pompe disease.</p></div></front></TEI><affiliations><list><country><li>États-Unis</li></country></list><tree><country name="États-Unis"><noRegion><name sortKey="Lim, Jeong A" sort="Lim, Jeong A" uniqKey="Lim J" first="Jeong-A" last="Lim">Jeong-A Lim</name></noRegion><name sortKey="Meena, Naresh Kumar" sort="Meena, Naresh Kumar" uniqKey="Meena N" first="Naresh Kumar" last="Meena">Naresh Kumar Meena</name><name sortKey="Raben, Nina" sort="Raben, Nina" uniqKey="Raben N" first="Nina" last="Raben">Nina Raben</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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