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Christian Mueller

genomic medicine unit

46H指数
157论文数
5.9K被引数
收录论文 83
发表时间
Taming immune responses to AAV gene therapy by programmed in vivo Treg expansion通过程序化体内Treg扩增来调控AAV基因疗法的免疫反应
err2026-02-13
err0
errOAAI
errLavesh Gwalani; Mincheol Park; Alexandra B. Ysasi; Mona Motwani; Robert Jackson; Jie Bu; Zhengyu Luo; John Bladon; Edith L. Pfister; Christian Mueller; Sourav R. Choudhury
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AAV gene therapy for GBA1 related diseasesAAV基因疗法用于GBA1相关疾病
err2025-12-09
err0
PREAI
errSwathi Ayloo; Jae Cheon Ryu; Shih-Ching Chou; Lydia Blatnik; Erik Wischhof; Jie Bu; Lilu Guo; Mahmud Hossain; Dhiman Ghosh; William McCarty; Ann Byrne; Lilly Chai; Jennifer Clarke; Can Kayatekin; Bailin Zhang; S.Pablo Sardi; Martin Goulet; Bradford Elmer; Christian Mueller; Shyam Ramachandran
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Artificial miRNA mediated reduction of SNCA for the treatment of α-synucleinopathies人工miRNA介导的SNCA降低用于α-突触核蛋白病的治疗
err2025-11-12
err0
PREAI
errBradford Elmer; Erik Wischhof; Tiffany Chan; Thanga Aishwarya Mahendran; Jeff Ardinger; Brenda Richards; Dongyu Liu; James Bladon; Ye He; Robert Jackson; Michael Fleming; Franck Rapaport; Shameer Khader; Martin Goulet; Christian Mueller; Shyam Ramachandran
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SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate EyeSNAC:一种非人灵长类眼睛中衣壳分布的单核体图谱
err2025-09-26
err0
PREAI
errRachna Manek; Eugenia Lyashenko; Andre H. Kurlovs; Yinyin Huang; Jeremy Huang; Margaret Hennessy; Jason Wu; Jasmine Bloom; Tess Torregrosa; Edith L. Pfister; Pavitra Ramachandran; Virginia Savova; Christian Mueller; Giorgio Gaglia; Sourav R. Choudhury
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Development of an AAV-delivered microRNA gene therapy for Myotonic Dystrophy Type 1开发一种AAV递送的microRNA基因疗法用于强直性肌营养不良症1型
err2025-09-02
err0
errOAAI
errGiulio S. Tomassy; Wei Fan; Shuwen Cao; Zhengyu Luo; Alessandro Magli; Tracy Zhang; Kristen Boyle; Robert Jackson; Brenda Richards; Dongyu Liu; Franck Rapaport; Shameer Khader; Edith L. Pfister; Catherine O’Riordan; Amy Frederick; Jennifer Sullivan; James Cao; Nageswara Rao Kollu; Basel T. Assaf; Martin Goulet; Christian Mueller
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AAV delivery of artificial miRNA targeting MAPT for the reduction of tau pathology in Alzheimer’s diseaseAAV介导的人工miRNA靶向MAPT,以减少阿尔茨海默病中的tau病理。
err2025-09-01
err0
PREAI
errJessica M. Hogestyn; Erik Wischhof; Yao Chen; Tiffany Chan; Brenda Richards; Thangaaishwarya Mahendran; Johanna Adams; Daniel Dubreuil; Jie Bu; Robert Jackson; Martin Goulet; Christian Mueller; Shyam Ramachandran; Bradford Elmer
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Reduction of Z alpha-1 antitrypsin polymers in human iPSC-hepatocytes and mice by LRRK2 inhibitors
err2024-07-02
err0
PREAI
errKent, Deniz; Ng, Soon Seng; Syanda, Adam M.; Khoshkenar, Payam; Ronzoni, Riccardo; Li, Chao Zheng; Zieger, Marina; Greer, Cindy; Hatch, Stephanie; Segal, Joe; Blackford, Samuel J., I; Im, Yu Ri; Chowdary, Vivek; Ismali, Taylor; Danovi, Davide; Lewis, Patrick A.; Irving, James A.; Sahdeo, Sunil; Lomas, David A.; Ebner, Daniel; Mueller, Christian; Rashid, S. Tamir
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AAV-ARSA mediated gene replacement for the treatment of metachromatic leukodystrophy
err2024-02-01
err0
PREAI
errRamachandran, Shyam; Ardinger, Jeffery; Ramos, MiAngela; Guo, Lilu; Hossain, Mahmud; Ayloo, Swathi; Adams, Johanna; McCarty, William; Nass, Shelley; Goulet, Martin; Mueller, Christian
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Evaluation of a novel AAV capsid with widespread CNS and peripheral biodistribution in non-human primates
err2024-02-01
err0
PREAI
errRamachandran, Shyam; Hogestyn, Jessica; Ayloo, Swathi; Ryu, Jae; Ardinger, Jeffery; Ramos, MiAngela; Blatnik, Lydia; Nass, Shelley; Goulet, Martin; Mueller, Christian; Gonzalez-Meneses, Antonio
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Comparison of edoxaban and enoxaparin in a rat model of AlCl3-induced thrombosis of the superior sagittal sinus
err2023-06-01
err2
errOAAI
errHachenberger, M.; Yeniguen, M.; Suenner, L.; Hinchliffe, D.; Mueller, C.; Wietelmann, A.; Gerriets, T.; Tschernatsch, M.; Juenemann, M.; Gerner, S. T.; Doeppner, T. R.; Huttner, H. B.; Braun, T.
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CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro
err2022-10-21
err30
errOAAI
errMeijboom, Katharina E.; Abdallah, Abbas; Fordham, Nicholas P.; Nagase, Hiroko; Rodriguez, Tomas; Kraus, Carolyn; Gendron, Tania F.; Krishnan, Gopinath; Esanov, Rustam; Andrade, Nadja S.; Rybin, Matthew J.; Ramic, Melina; Stephens, Zachary D.; Edraki, Alireza; Blackwood, Meghan T.; Kahriman, Aydan; Henninger, Nils; Kocher, Jean-Pierre A.; Benatar, Michael; Brodsky, Michael H.; Petrucelli, Leonard; Gao, Fen-Biao; Sontheimer, Erik J.; Brown, Robert H.; Zeier, Zane; Mueller, Christian
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Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates
err2022-05-04
err13
errOAAI
errMilani, Michela; Canepari, Cesare; Liu, Tongyao; Biffi, Mauro; Russo, Fabio; Plati, Tiziana; Curto, Rosalia; Patarroyo-White, Susannah; Drager, Douglas; Visigalli, Ilaria; Brombin, Chiara; Albertini, Paola; Follenzi, Antonia; Ayuso, Eduard; Mueller, Christian; Annoni, Andrea; Naldini, Luigi; Cantore, Alessio
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Evaluation of cytosine base editing and adenine base editing as a potential treatment for alpha-1 antitrypsin deficiency
err2022-04-01
err22
errOAAI
errPacker, Michael S.; Chowdhary, Vivek; Lung, Genesis; Cheng, Lo-, I; Aratyn-Schaus, Yvonne; Leboeuf, Dominique; Smith, Sarah; Shah, Aalok; Chen, Delai; Zieger, Marina; Cafferty, Brian J.; Yan, Bo; Ciaramella, Giuseppe; Gregoire, Francine M.; Mueller, Christian
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Ferret models of alpha-1 antitrypsin deficiency develop lung and liver disease
err2022-03-08
err15
errOAAI
errHe, Nan; Liu, Xiaoming; Vegter, Amber R.; Evans, T. Idil A.; Gray, Jaimie S.; Guo, Junfeng; Moll, Shashanna R.; Guo, Lydia J.; Luo, Meihui; Ma, Ningxia; Sun, Xingshen; Liang, Bo; Yan, Ziying; Feng, Zehua; Qi, Lisi; Joshi, Arnav S.; Shahin, Weam; Yi, Yaling; Gibson-Corley, Katherine N.; Hoffman, Eric A.; Wang, Kai; Mueller, Christian; Engelhardt, John F.; Rosen, Bradley H.
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AAV gene therapy for Tay-Sachs disease
err2022-02-10
err67
errOAAI
errFlotte, Terence R.; Cataltepe, Oguz; Puri, Ajit; Batista, Ana Rita; Moser, Richard; McKenna-Yasek, Diane; Douthwright, Catherine; Gernoux, Gwladys; Blackwood, Meghan; Mueller, Christian; Tai, Phillip W. L.; Jiang, Xuntian; Bateman, Scot; Spanakis, Spiro G.; Parzych, Julia; Keeler, Allison M.; Abayazeed, Aly; Rohatgi, Saurabh; Gibson, Laura; Finberg, Robert; Barton, Bruce A.; Vardar, Zeynep; Shazeeb, Mohammed Salman; Gounis, Matthew; Tifft, Cynthia J.; Eichler, Florian S.; Brown, Robert H., Jr.; Martin, Douglas R.; Gray-Edwards, Heather L.; Sena-Esteves, Miguel
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SepA Enhances Shigella Invasion of Epithelial Cells by Degrading Alpha-1 Antitrypsin and Producing a Neutrophil Chemoattractant
errMBIO
IF4.7
err2021-12-21
err12
errOAAI
errMeza-Segura, Mario; Birtley, James R.; Maldonado-Contreras, Ana; Mueller, Christian; Simin, Karl J.; Stern, Lawrence J.; McCormick, Beth A.
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Hypoimmune induced pluripotent stem cell-derived cell therapeutics treat cardiovascular and pulmonary diseases in immunocompetent allogeneic mice
err2021-07-09
err27
errOAAI
errDeuse, Tobias; Tediashvili, Grigol; Hu, Xiaomeng; Gravina, Alessia; Tamenang, Annika; Wang, Dong; Connolly, Andrew; Mueller, Christian; Mallavia, Benat; Looney, Mark R.; Alawi, Malik; Lanier, Lewis L.; Schrepfer, Sonja
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Genetic diversity of axon degenerative mechanisms in models of Parkinson's disease帕金森病模型轴突退变机制的遗传多样性
err2021-07-01
err20
errOAAI
errPeters, Owen M.; Weiss, Alexandra; Metterville, Jake; Song, Lina; Logan, Robert; Smith, Gaynor A.; Schwarzschild, Michael A.; Mueller, Christian; Brown, Robert H.; Freeman, Marc
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