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Res."},{"key":"ref51\/cit51","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1002\/cbic.200700496","volume":"9","author":"Li H.","year":"2008","journal-title":"ChemBioChem"},{"key":"ref52\/cit52","doi-asserted-by":"crossref","first-page":"1109","DOI":"10.1007\/s10719-009-9231-3","volume":"26","author":"Butters T. D.","year":"2009","journal-title":"Glycoconjugate J."},{"key":"ref53\/cit53","doi-asserted-by":"crossref","first-page":"5390","DOI":"10.1021\/ja809776r","volume":"131","author":"Marcelo F.","year":"2009","journal-title":"J. Am. Chem. Soc."},{"key":"ref54\/cit54","doi-asserted-by":"crossref","first-page":"535","DOI":"10.1055\/s-2001-12340","volume":"4","author":"Xiao X.","year":"2001","journal-title":"Synlett"},{"key":"ref55\/cit55","doi-asserted-by":"crossref","first-page":"5958","DOI":"10.1021\/jo00123a037","volume":"60","author":"Lohray B. B.","year":"1995","journal-title":"J. Org. Chem."},{"key":"ref56\/cit56","doi-asserted-by":"crossref","first-page":"248","DOI":"10.1016\/0003-2697(76)90527-3","volume":"72","author":"Bradford M. M.","year":"1976","journal-title":"Anal. Biochem."},{"key":"ref57\/cit57","doi-asserted-by":"crossref","first-page":"e16347","DOI":"10.1371\/journal.pone.0016347","volume":"6","author":"Orwig S. D.","year":"2011","journal-title":"PLoS One"},{"key":"ref58\/cit58","doi-asserted-by":"crossref","first-page":"307","DOI":"10.1016\/S0076-6879(97)76066-X","volume":"276","author":"Otwinowski Z.","year":"1997","journal-title":"Methods Enzymol."},{"key":"ref59\/cit59","first-page":"760","volume":"50","author":"Collaborative Computational Project Number 4","year":"1994","journal-title":"Acta Crystallogr."},{"key":"ref60\/cit60","first-page":"486","volume":"66","author":"Emsley P.","year":"2010","journal-title":"Acta Crystallogr."},{"key":"ref61\/cit61","first-page":"1355","volume":"60","author":"Schuttelkopf A. 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