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Chem."},{"key":"key\n\t\t\t\t20170522150843_DDU349C84","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1016\/j.cbi.2007.01.015","article-title":"Nanoconjugate based on polymalic acid for tumor targeting","volume":"171","year":"2008","journal-title":"Chem. Biol. Interact."},{"key":"key\n\t\t\t\t20170522150843_DDU349C85","doi-asserted-by":"crossref","first-page":"2366","DOI":"10.1073\/pnas.1221891110","article-title":"Aberrant splicing of HTT generates the pathogenic exon 1 protein in Huntington disease","volume":"110","year":"2013","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"key\n\t\t\t\t20170522150843_DDU349C86","doi-asserted-by":"crossref","first-page":"221","DOI":"10.1007\/978-1-59745-019-5_16","article-title":"The preparation of primary cortical neuron cultures and a practical application using immunofluorescent cytochemistry","volume":"633","year":"2010","journal-title":"Methods Mol. 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