Search Results - Elizabeth M. Simpson
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Human progranulin-expressing mice as a novel tool for the development of progranulin-modulating therapeutics by Terri L. Petkau, Benjamin Life, Ge Lu, Jasmine Yang, Oriol Fornes, Wyeth Wasserman, Elizabeth M. Simpson, Blair R. Leavitt
Published in Neurobiology of Disease (2021-06-01)Get full text
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Germline CRISPR/Cas9-Mediated Gene Editing Prevents Vision Loss in a Novel Mouse Model of Aniridia by 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
Published in Molecular Therapy: Methods & Clinical Development (2020-06-01)Get full text
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ABE8e Corrects Pax6-Aniridic Variant in Humanized Mouse ESCs and via LNPs in Ex Vivo Cortical Neurons by 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
Published in Ophthalmology and Therapy (2023-05-01)Get full text
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Taf1 knockout is lethal in embryonic male mice and heterozygous females show weight and movement disorders by 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
Published in Disease Models & Mechanisms (2024-07-01)Get full text
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New MiniPromoter Ple389 (ADORA2A) drives selective expression in medium spiny neurons in mice and non-human primates by 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
Published in Scientific Reports (2024-11-01)Get full text
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Targeted CNS delivery using human MiniPromoters and demonstrated compatibility with adeno-associated viral vectors by 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
Published in Molecular Therapy: Methods & Clinical Development (2014-01-01)Get full text
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