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Publications

GENE THERAPY FOR CNS DISORDERS

Adeno-associated virus serotype 1-based gene therapy for FTD caused by GRN mutations | September 16, 2020
Christian Hinderer, Rod Miller, Cecilia Dyer, Julia Johansson, Peter Bell, Elizabeth Buza, James M. Wilson

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A Single Injection of an Optimized Adeno-Associated Viral Vector into Cerebrospinal Fluid Corrects Neurological Disease in a Murine Model of GM1 Gangliosidosis | November 13, 2020
Christian Hinderer, Brenden Nosratbakhsh, Nathan Katz, James M. Wilson

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Gene Therapy for Treatment of Monogenic Neurodegenerative Diseases | April 2021
Karen M. Anderson, D. Elizabeth McNeil, David A. Weinstein, Gary Romano

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OTHER GENE THERAPY TOPICS

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals | August 14, 2017
Christian Hinderer, Peter Bell, Nathan Katz, Charles H. Vite, Jean-Pierre Louboutin, Erin Bote, Hongwei Yu, Yanqing Zhu, Margret L. Casal, Jessica Bagel, Patricia O’Donnell, Ping Wang, Mark E. Haskins, Tamara Goode, James M. Wilson

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Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology | June 25, 2020
Juliette Hordeaux, Elizabeth L. Buza, Cecilia Dyer, Tamara Goode, Thomas W. Mitchell, Laura Richman, Nathan Denton, Christian Hinderer, Nathan Katz, Ralf Schmid, Rod Miller, Gourav R. Choudhury, Makoto Horiuchi, Kalyani Nambiar, Hanying Yan, Mingyao Li, James M. Wilson

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MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates | November 11, 2020
Juliette Hordeaux, Elizabeth L. Buza, Brianne Jeffrey, Chunjuan Song, Tahsin Jahan, Yuan Yuan, Yanqing Zhu, Peter Bell, Mingyao Li, Jessica A. Chichester, Roberto Calcedo, James M. Wilson

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