Pros and cons of different ways to address dysfunctional autophagy in Pompe disease
Identifieur interne : 000468 ( Pmc/Checkpoint ); précédent : 000467; suivant : 000469Pros 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><pmc article-type="review-article"><pmc-comment>The publisher of this article does not allow downloading of the full text in XML form.</pmc-comment>
<front><journal-meta><journal-id journal-id-type="nlm-ta">Ann Transl Med</journal-id><journal-id journal-id-type="iso-abbrev">Ann Transl Med</journal-id><journal-id journal-id-type="publisher-id">ATM</journal-id><journal-title-group><journal-title>Annals of Translational Medicine</journal-title></journal-title-group><issn pub-type="ppub">2305-5839</issn><issn pub-type="epub">2305-5847</issn><publisher><publisher-name>AME Publishing Company</publisher-name></publisher></journal-meta><article-meta><article-id pub-id-type="pmid">31392191</article-id><article-id pub-id-type="pmc">6642936</article-id><article-id pub-id-type="publisher-id">atm-07-13-279</article-id><article-id pub-id-type="doi">10.21037/atm.2019.03.51</article-id><article-categories><subj-group subj-group-type="heading"><subject>Review Article</subject></subj-group></article-categories><title-group><article-title>Pros and cons of different ways to address dysfunctional autophagy in Pompe disease</article-title></title-group><contrib-group><contrib contrib-type="author"><name><surname>Lim</surname><given-names>Jeong-A</given-names></name><xref ref-type="aff" rid="aff1"><sup>1</sup></xref><xref ref-type="author-notes" rid="afn2"><sup>#</sup></xref></contrib><contrib contrib-type="author"><name><surname>Meena</surname><given-names>Naresh Kumar</given-names></name><xref ref-type="aff" rid="aff2"><sup>2</sup></xref><xref ref-type="author-notes" rid="afn2"><sup>#</sup></xref></contrib><contrib contrib-type="author" corresp="yes"><name><surname>Raben</surname><given-names>Nina</given-names></name><xref ref-type="aff" rid="aff2"><sup>2</sup></xref></contrib><aff id="aff1"><label>1</label>Division of Medical Genetics, Department of Pediatrics,<institution>Duke University School of Medicine</institution>,<addr-line>Durham, NC</addr-line>,<country>USA</country>;</aff><aff id="aff2"><label>2</label>Cell Biology and Physiology Center, National Heart, Lung, and Blood Institute, NIH,<addr-line>Bethesda, MD</addr-line>,<country>USA</country></aff></contrib-group><author-notes><fn id="afn1"><p><italic>Contributions:</italic> (I) Conception and design: N Raben; (II) Administrative support: None; (III) Provision of study materials or patients: N Raben; (IV) Collection and assembly of data: All authors; (V) Data analysis and interpretation: JA Lim, NK Meena; (VI) Manuscript writing: All authors; (VII) Final approval of manuscript: All authors.</p></fn><fn id="afn2" fn-type="equal"><label>#</label><p>These authors contributed equally to this work.</p></fn><corresp id="cor1"><italic>Correspondence to:</italic> Nina Raben. Cell Biology and Physiology Center, National Heart, Lung, and Blood Institute, NIH, Bethesda, MD, USA. Email: <email xlink:href="rabenn@mail.nih.gov">rabenn@mail.nih.gov</email>.</corresp><fn fn-type="COI-statement"><p><italic>Conflicts of Interest:</italic> The authors have no conflicts of interest to declare.</p></fn></author-notes><pub-date pub-type="epub-ppub"><month>7</month><year>2019</year></pub-date><pmc-comment>Fake ppub date generated by PMC from publisher
pub-date/@pub-type='epub-ppub' </pmc-comment>
<pub-date pub-type="ppub"><month>7</month><year>2019</year></pub-date><volume>7</volume><issue>13</issue><elocation-id>279</elocation-id><history><date date-type="received"><day>19</day><month>3</month><year>2019</year></date><date date-type="accepted"><day>20</day><month>3</month><year>2019</year></date></history><permissions><copyright-statement>2019 Annals of Translational Medicine. All rights reserved.</copyright-statement><copyright-year>2019</copyright-year><copyright-holder>Annals of Translational Medicine.</copyright-holder></permissions><abstract><p>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.</p></abstract><kwd-group kwd-group-type="author"><title>Keywords: </title><kwd>Lysosome</kwd><kwd>autophagy</kwd><kwd>enzyme replacement therapy (ERT)</kwd><kwd>muscle</kwd></kwd-group></article-meta></front></pmc><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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