Recent Publications

Cas9 Editing of ATXN1 in a Spinocerebellar Ataxia type 1 Mice and human iPSC-derived neurons

Kelly J. Fagan, Guillem Chillon, Ellie M. Carrell, Elisa A. Waxman, Beverly L. Davidson Moleculary Therapy Nucleic Acids. 2024 August 30

DOI: https://doi.org/10.1016/j.omtn.2024.102317

Optimized AAV capsids for diseases of the basal ganglia show robust potency and distribution in adult nonhuman primates

David E. Leib, Yonghong Chen, Luis Tecedor, Paul T. Ranum, Megan S. Keiser, Bryan C. Lewandowski, Ellie M. Carrell, Sakshi Arora, Icnelia Huerta Ocampo, Xueyuan Liu, Beverly L Davidson bioRxiv. 2024 May 2

DOI: https://doi.org/10.1101/2024.05.02.592211

Temporal restriction of Cas9 expression improves CRISPR-mediated deletion efficacy and fidelity

Jesse A. Weber, Jonathan F. Lang, Ellie M. Carrell, Mohamad-Gabriel Alameh, Beverly L. Davidson Mol. Therapy. Nucleic Acids. 2024 March 11

DOI: https://doi.org/10.1016/j.omtn.2024.102172

APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease

Rosemary J. Jackson, Megan S. Keiser, Jonah C. Meltzer, Dustin P. Fykstra, Steven E. Dierksmeier, Soroush Hajizadeh, Johannes Kreuzer, Robert Morris, Alexandra Melloni, Tsuneo Nakajima, Luis Tecedor, Paul T. Ranum, Ellie Carrell, YongHong Chen, Maryam A. Nishtar, David M. Holtzman, Wilhelm Haas, Beverly L. Davidson, Bradley T. Hyman Mol. Therapy. 2024 March 19

DOI: https://doi.org/10.1016/j.ymthe.2024.03.024

AAV-based delivery of RNAi targeting Ataxin-2 improves survival, strength, and pathology in mouse models of rapidly and slowly progressive sporadic ALS

Defne A. Amado, Ashley B. Robbins, Alicia R. Smith, Katherine R. Whiteman, Guillem Chillon Bosch, Yonghong Chen, Joshua A. Fuller, Aleksandar Izda, Shareen Nelson, Abigail I. Dichter, Alex Mas Monteys, Beverly L. Davidson bioRxiv (Preprint). 2024 Feb 2

DOI: https://doi.org/10.1101/2024.01.31.578314

A novel iPSC model reveals selective vulnerability of neurons in multiple sulfatase deficiency

Vi Pham, Livia Sertori Finoti, Margaret M Cassidy, Jean Ann Maguire, Alyssa L Gagne, Elisa A Waxman, Deborah L French, Kaitlyn King, Zitao Zhou, Michael H Gelb, Parith Wongkittichote, Xinying Hong, Lars Schlotawa, Beverly L Davidson, Rebecca C Ahrens-Nicklas Mol. Genet Metab. 2024 Feb

DOI: https://doi.org/10.1016/j.ymgme.2023.108116