Rezultati - Elizabeth M Simpson

Modelling human regulatory variation in mouse: finding the function in genome-wide association studies and whole-genome sequencing. od Jean-François Schmouth, Russell J Bonaguro, Ximena Corso-Diaz, Elizabeth M Simpson

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SAGE2Splice: unmapped SAGE tags reveal novel splice junctions. od Byron Yu-Lin Kuo, Ying Chen, Slavita Bohacec, Ojvind Johansson, Wyeth W Wasserman, Elizabeth M Simpson

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Human progranulin-expressing mice as a novel tool for the development of progranulin-modulating therapeutics od Terri L. Petkau, Benjamin Life, Ge Lu, Jasmine Yang, Oriol Fornes, Wyeth Wasserman, Elizabeth M. Simpson, Blair R. Leavitt

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Transgene distribution and immune response after ultrasound delivery of rAAV9 and PHP.B to the brain in a mouse model of amyloidosis od Rikke Hahn Kofoed, Stefan Heinen, Joseph Silburt, Sonam Dubey, Chinaza Lilian Dibia, Miriam Maes, Elizabeth M. Simpson, Kullervo Hynynen, Isabelle Aubert

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Germline CRISPR/Cas9-Mediated Gene Editing Prevents Vision Loss in a Novel Mouse Model of Aniridia od Seyedeh Zeinab Mirjalili Mohanna, Jack W. Hickmott, Siu Ling Lam, Nina Y. Chiu, Tess C. Lengyell, Beatrice M. Tam, Orson L. Moritz, Elizabeth M. Simpson

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ABE8e Corrects Pax6-Aniridic Variant in Humanized Mouse ESCs and via LNPs in Ex Vivo Cortical Neurons od Bethany A. Adair, Andrea J. Korecki, Diana Djaksigulova, Pamela K. Wagner, Nina Y. Chiu, Siu Ling Lam, Tess C. Lengyell, Blair R. Leavitt, Elizabeth M. Simpson

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Humanized mice carrying a pathogenic GRN deletion as a pre-clinical platform for targeted gene therapies in frontotemporal dementia od Benjamin E. Life, Pardis Kazemian, Terri L. Petkau, Alissandra de Moura Gomes, Sarah B. Thomson, Ning Shen, Junia Myung, Farah Jayousi, Tess C. Lengyell, Philipp F. Lange, Elizabeth M. Simpson, Keegan Korthauer, Blair R. Leavitt

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Taf1 knockout is lethal in embryonic male mice and heterozygous females show weight and movement disorders od Elisa M. Crombie, Andrea J. Korecki, Karen Cleverley, Bethany A. Adair, Thomas J. Cunningham, Weaverly Colleen Lee, Tess C. Lengyell, Cheryl Maduro, Victor Mo, Liam M. Slade, Ines Zouhair, Elizabeth M. C. Fisher, Elizabeth M. Simpson

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New MiniPromoter Ple389 (ADORA2A) drives selective expression in medium spiny neurons in mice and non-human primates od Alissandra de Moura Gomes, Terri L. Petkau, Andrea J. Korecki, Oriol Fornes, Adriana Galvan, Ge Lu, Austin M. Hill, Siu Ling Lam, Anqi Yao, Rachelle A. Farkas, Wyeth W. Wasserman, Yoland Smith, Elizabeth M. Simpson, Blair R. Leavitt

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MiniPromoters Ple384 (TH) and Ple388 (PITX3) for targeting midbrain dopaminergic neurons in mice and monkeys od Adriana Galvan, Diane Choi, Andrea J. Korecki, Alissandra de Moura Gomes, Oriol Fornes, Jun Tanimura, Rachelle A. Farkas, Siu Ling Lam, Ge Lu, Terri L. Petkau, Anqi Yao, Wyeth W. Wasserman, Blair R. Leavitt, Elizabeth M. Simpson, Yoland Smith

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PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina od Jack W Hickmott, Chih-yu Chen, David J Arenillas, Andrea J Korecki, Siu Ling Lam, Laurie L Molday, Russell J Bonaguro, Michelle Zhou, Alice Y Chou, Anthony Mathelier, Sanford L Boye, William W Hauswirth, Robert S Molday, Wyeth W Wasserman, Elizabeth M Simpson

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HMMR acts in the PLK1-dependent spindle positioning pathway and supports neural development od Marisa Connell, Helen Chen, Jihong Jiang, Chia-Wei Kuan, Abbas Fotovati, Tony LH Chu, Zhengcheng He, Tess C Lengyell, Huaibiao Li, Torsten Kroll, Amanda M Li, Daniel Goldowitz, Lucien Frappart, Aspasia Ploubidou, Millan S Patel, Linda M Pilarski, Elizabeth M Simpson, Philipp F Lange, Douglas W Allan, Christopher A Maxwell

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Targeted CNS delivery using human MiniPromoters and demonstrated compatibility with adeno-associated viral vectors od Charles N de Leeuw, Frank M Dyka, Sanford L Boye, Stéphanie Laprise, Michelle Zhou, Alice Y Chou, Lisa Borretta, Simone C McInerny, Kathleen G Banks, Elodie Portales-Casamar, Magdalena I Swanson, Cletus A D'Souza, Shannon E Boye, Steven JM Jones, Robert A Holt, Daniel Goldowitz, William W Hauswirth, Wyeth W Wasserman, Elizabeth M Simpson

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