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Inherited Metab. Dis."},{"key":"ref17\/cit17","first-page":"472","volume":"4","author":"Lachmann R. H.","year":"2003","journal-title":"Curr. Opin. Invest. Drugs"},{"key":"ref18\/cit18","doi-asserted-by":"crossref","first-page":"1481","DOI":"10.1016\/S0140-6736(00)02161-9","volume":"355","author":"Cox T.","year":"2000","journal-title":"Lancet"},{"key":"ref19\/cit19","doi-asserted-by":"crossref","first-page":"757","DOI":"10.1023\/B:BOLI.0000045756.54006.17","volume":"27","author":"Elstein D.","year":"2004","journal-title":"J. Inherited Metab. 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Med."},{"key":"ref25\/cit25","doi-asserted-by":"crossref","first-page":"821","DOI":"10.1111\/j.1600-0854.2004.00232.x","volume":"5","author":"Ulloa-Aguirre A.","year":"2004","journal-title":"Traffic"},{"key":"ref26\/cit26","doi-asserted-by":"crossref","first-page":"1677","DOI":"10.1016\/j.bbapap.2006.08.012","volume":"1764","author":"Arakawa T.","year":"2006","journal-title":"Biochim. Biophys. Acta"},{"key":"ref27\/cit27","doi-asserted-by":"crossref","first-page":"901","DOI":"10.2174\/138955708785132783","volume":"8","author":"Leandro P.","year":"2008","journal-title":"Mini-Rev. Med. Chem."},{"key":"ref28\/cit28","doi-asserted-by":"crossref","first-page":"1882","DOI":"10.1126\/science.286.5446.1882","volume":"286","author":"Ellgaard L.","year":"1999","journal-title":"Science"},{"key":"ref29\/cit29","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1038\/nrm1052","volume":"4","author":"Ellgaard L.","year":"2003","journal-title":"Nat. Rev. Mol. 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Biol."},{"key":"ref35\/cit35","doi-asserted-by":"crossref","first-page":"13813","DOI":"10.1073\/pnas.0605928103","volume":"103","author":"Steet R. A.","year":"2006","journal-title":"Proc. Natl. Acad. Sci. U.S.A."},{"key":"ref36\/cit36","doi-asserted-by":"crossref","first-page":"424","DOI":"10.1007\/s10545-009-1077-0","volume":"32","author":"Benjamin E. R.","year":"2009","journal-title":"J. Inherited Metab. Dis."},{"key":"ref37\/cit37","doi-asserted-by":"crossref","first-page":"3790","DOI":"10.1016\/j.bmc.2010.04.048","volume":"18","author":"Kato A.","year":"2010","journal-title":"Bioorg. Med. Chem."},{"key":"ref38\/cit38","doi-asserted-by":"crossref","first-page":"101","DOI":"10.1038\/nchembio850","volume":"3","author":"Lieberman R. L.","year":"2007","journal-title":"Nat. Chem. Biol."},{"key":"ref39\/cit39","doi-asserted-by":"crossref","first-page":"2643","DOI":"10.1002\/cbic.200800249","volume":"9","author":"Kornhaber G. 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Res."},{"key":"ref49\/cit49","doi-asserted-by":"crossref","first-page":"771","DOI":"10.1517\/17425247.3.6.771","volume":"3","author":"Grabowski G. A.","year":"2006","journal-title":"Expert Opin. Drug Delivery"},{"key":"ref50\/cit50","doi-asserted-by":"crossref","first-page":"4816","DOI":"10.1021\/bi9002265","volume":"48","author":"Lieberman R. L.","year":"2009","journal-title":"Biochemistry"},{"key":"ref51\/cit51","doi-asserted-by":"crossref","first-page":"5258","DOI":"10.1021\/bi048135v","volume":"44","author":"Matulis D.","year":"2005","journal-title":"Biochemistry"},{"key":"ref52\/cit52","doi-asserted-by":"crossref","first-page":"13067","DOI":"10.1016\/S0021-9258(17)38839-7","volume":"260","author":"Basu A.","year":"1985","journal-title":"J. Biol. Chem."},{"key":"ref53\/cit53","doi-asserted-by":"crossref","first-page":"76","DOI":"10.1016\/0167-4838(86)90010-5","volume":"870","author":"Choy F. Y","year":"1986","journal-title":"Biochim. Biophys. 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