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基因编辑仓鼠Reduction of TMEM97 increases NPC1 protein levels and restores cholesterol trafficking in Niemann-pick type C1 disease cells.

期刊:Human molecular genetics发表年份:2016作者:Darius Ebrahimi-Fakhari; Lara Wahlster; Fabian Bartz; Jennifer Werenbeck-Ueding; Maria Praggastis; Jessie Zhang; Brigitte Joggerst-Thomalla; Susanne Theiss; Dirk Grimm; Daniel S Ory; Heiko Runz

基因编辑仓鼠Optimization and in Vivo Validation of Peptide Vectors Targeting the LDL Receptor.

期刊:Molecular pharmaceutics发表年份:2016作者:Guillaume Jacquot; Pascaline Lécorché; Jean-Daniel Malcor; Mathieu Laurencin; Maria Smirnova; Karine Varini; Cédric Malicet; Fanny Gassiot; Karima Abouzid; Aude Faucon; Marion David; Nicolas Gaudin; Maxime Masse; Géraldine Ferracci; Vincent Dive; Sal

基因编辑仓鼠Syncytial Mutations Do Not Impair the Specificity of Entry and Spread of a Glycoprotein D Receptor-Retargeted Herpes Simplex Virus.

期刊:Journal of virology发表年份:2016作者:Yu Okubo; Hiroaki Uchida; Aika Wakata; Takuma Suzuki; Tomoko Shibata; Hitomi Ikeda; Miki Yamaguchi; Justus B Cohen; Joseph C Glorioso; Mitsuo Tagaya; Hirofumi Hamada; Hideaki Tahara

基因编辑仓鼠Early-onset epileptic encephalopathy caused by a reduced sensitivity of Kv7.2 potassium channels to phosphatidylinositol 4,5-bisphosphate.

期刊:Scientific reports发表年份:2016作者:Maria Virginia Soldovieri; Paolo Ambrosino; Ilaria Mosca; Michela De Maria; Edoardo Moretto; Francesco Miceli; Alessandro Alaimo; Nunzio Iraci; Laura Manocchio; Alessandro Medoro; Maria Passafaro; Maurizio Taglialatela