{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T12:14:20Z","timestamp":1791980060629,"version":"4.4.0"},"reference-count":30,"publisher":"American Association for the Advancement of Science (AAAS)","issue":"6386","funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["HL135736"],"award-info":[{"award-number":["HL135736"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["HL128071"],"award-info":[{"award-number":["HL128071"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Science"],"published-print":{"date-parts":[[2018,4,20]]},"abstract":"<jats:title>An innovative approach for a rare disease<\/jats:title>\n                  <jats:p>\n                    Charcot-Marie-Tooth disease type 2A (CMT2A) is a rare, inherited neurodegenerative condition. Affected individuals develop severe progressive muscle weakness, motor deficits, and peripheral neuropathy. Although defects in the gene encoding mitofusin 2 (MFN2) are known to cause CMT2A, the disease remains incurable. Rocha\n                    <jats:italic>et al.<\/jats:italic>\n                    identified specific MFN2 residues contributing to the disease and developed a class of MFN2-agonist drugs. The small molecules restored mitochondrial fusion and activity in the sciatic nerves of mice; they may also help in other diseases linked to mitochondrial trafficking.\n                  <\/jats:p>\n                  <jats:p>\n                    <jats:italic>Science<\/jats:italic>\n                    , this issue p.\n                    <jats:related-article xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"doi\" issue=\"6386\" page=\"336\" related-article-type=\"in-this-issue\" vol=\"360\" xlink:href=\"10.1126\/science.aao1785\">336<\/jats:related-article>\n                  <\/jats:p>","DOI":"10.1126\/science.aao1785","type":"journal-article","created":{"date-parts":[[2018,5,7]],"date-time":"2018-05-07T15:16:17Z","timestamp":1525706177000},"page":"336-341","source":"Crossref","is-referenced-by-count":246,"title":["MFN2 agonists reverse mitochondrial defects in preclinical models of Charcot-Marie-Tooth disease type 2A"],"prefix":"10.1126","volume":"360","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7134-7376","authenticated-orcid":true,"given":"Agostinho G.","family":"Rocha","sequence":"first","affiliation":[{"name":"Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5487-1800","authenticated-orcid":true,"given":"Antonietta","family":"Franco","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3246-5008","authenticated-orcid":true,"given":"Andrzej M.","family":"Krezel","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Biophysics, Washington University School of Medicine, St. Louis, MO, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeanne M.","family":"Rumsey","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Justin M.","family":"Alberti","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"William C.","family":"Knight","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4055-5521","authenticated-orcid":true,"given":"Nikolaos","family":"Biris","sequence":"additional","affiliation":[{"name":"Departments of Biochemistry and Medicine, Wilf Family Cardiovascular Research Institute, Albert Einstein Cancer Center, Albert Einstein College of Medicine, Bronx, NY, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emmanouil","family":"Zacharioudakis","sequence":"additional","affiliation":[{"name":"Departments of Biochemistry and Medicine, Wilf Family Cardiovascular Research Institute, Albert Einstein Cancer Center, Albert Einstein College of Medicine, Bronx, NY, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9888-5411","authenticated-orcid":true,"given":"James W.","family":"Janetka","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Biophysics, Washington University School of Medicine, St. Louis, MO, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0100-8376","authenticated-orcid":true,"given":"Robert H.","family":"Baloh","sequence":"additional","affiliation":[{"name":"Department of Neurology, Cedars-Sinai Medical Center, Los Angeles, CA, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1395-9694","authenticated-orcid":true,"given":"Richard N.","family":"Kitsis","sequence":"additional","affiliation":[{"name":"Departments of Medicine and Cell Biology, Wilf Family Cardiovascular Research Institute, Albert Einstein Cancer Center, and Einstein-Mount Sinai Diabetes Research Center, Albert Einstein College of Medicine, Bronx, NY, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6691-8733","authenticated-orcid":true,"given":"Daria","family":"Mochly-Rosen","sequence":"additional","affiliation":[{"name":"Department of Chemical and Systems Biology, Stanford University School of Medicine, Stanford, CA, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R. 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