Publications by authors named "Beverly Davidson"

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The future of gene therapy: Safer vectors, sharper focus: High-profile failures demand deep root cause analysis - but the transformative potential of AAV remains within reach if the field is willing to learn and evolve.
Jeffrey S Chamberlain, Beverly L Davidson, Lindsey A George, Barry J Byrne, David Barrett

Mol Ther· October 2025

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Author Correction: AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice.
Defne A Amado, Ashley B Robbins, Katherine R Whiteman, Alicia R Smith, Guillem Chillon, Beverly L Davidson

Nat Commun· October 2025

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CRISPR-mediated transcriptional activation as a mutation-independent therapeutic strategy for -related intellectual disability.
Laura Sichlinger, Molly B Reilly, Sakshi Arora, Shuo Zhang, Nicolas Marotta, Beverly L Davidson

bioRxiv· October 2025

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Co-Opting MBNL-Dependent Alternative Splicing Cassette Exons to Control Gene Therapy in Myotonic Dystrophy.
Samuel T Carrell, Ellie M Carrell, Ryan Giovenco, Beverly L Davidson

Ann Neurol· August 2025

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AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice.
Defne A Amado, Ashley B Robbins, Katherine R Whiteman, Alicia R Smith, Guillem Chillon, Beverly L Davidson

Nat Commun· June 2025

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Current trends in gene therapy to treat inherited disorders of the brain.
Zaneta Matuszek, Brandon L Brown, Carolyn M Yrigollen, Megan S Keiser, Beverly L Davidson

Mol Ther· May 2025

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An AAV variant selected through NHP screens robustly transduces the brain and drives secreted protein expression in NHPs and mice.
Luis Tecedor, Yong Hong Chen, David E Leib, Paul T Ranum, Megan S Keiser, Beverly L Davidson

Sci Transl Med· May 2025

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Scouring the human Hsp70 network uncovers diverse chaperone safeguards buffering TDP-43 toxicity.
Edward M Barbieri, Miriam Linsenmeier, Katherine R Whiteman, Yan Cheng, Sylvanne Braganza, Beverly L Davidson

bioRxiv· May 2025

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Lipid Nanoparticles for Lung Delivery of CRISPR-Cas9 Ribonucleoproteins Allow Gene Editing of Clinical Targets.
Rebecca M Haley, Marshall S Padilla, Rakan D El-Mayta, Ryann A Joseph, Jesse A Weber, Beverly L Davidson

ACS Nano· April 2025

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CRISPR screen reveals modifiers of rAAV production including known rAAV infection genes playing an unexpected role in vector production.
Emily E O'Driscoll, Sakshi Arora, Jonathan F Lang, Beverly L Davidson, Ophir Shalem

Mol Ther Methods Clin Dev· March 2025

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intronic expansion identified by poly-glycine-arginine pathology increases Alzheimer's disease risk.
Lien Nguyen, Ramadan Ajredini, Shu Guo, Lisa E L Romano, Rodrigo F Tomas, Beverly L Davidson

Proc Natl Acad Sci U S A· February 2025

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Cas9 editing of 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

Mol Ther Nucleic Acids· December 2024

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Evaluation of a rapid multi-attribute combinatorial high-throughput UV-Vis/DLS/SLS analytical platform for rAAV quantification and characterization.
Xueyuan Liu, Riffard Jean-Gilles, Julia Baginski, Christina Cai, Ruilan Yan, Beverly L Davidson

Mol Ther Methods Clin Dev· September 2024

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CRISPR screen for rAAV production implicates genes associated with infection.
Emily E O'Driscoll, Sakshi Arora, Jonathan F Lang, Beverly L Davidson, Ophir Shalem

bioRxiv· September 2024

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Generation of humanized mouse models to support therapeutic development for SYNGAP1 and STXBP1 disorders.
Alex J Felix, Taryn Wilson, Rani Randell, Nicolas Marotta, Keita Uchida, Beverly L Davidson

bioRxiv· August 2024

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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 Ther Nucleic Acids· June 2024

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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, Beverly L Davidson

Mol Ther· May 2024

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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, Beverly L Davidson

Mol Genet Metab· February 2024

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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, Beverly L Davidson

bioRxiv· February 2024

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Antagonistic roles of canonical and Alternative-RPA in disease-associated tandem CAG repeat instability.
Terence Gall-Duncan, Jennifer Luo, Carla-Marie Jurkovic, Laura A Fischer, Kyota Fujita, Beverly L Davidson

Cell· October 2023

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-derived ependyma promoter drives increased therapeutic protein secretion into the CSF.
Ellie M Carrell, Yong Hong Chen, Paul T Ranum, Stephanie L Coffin, Larry N Singh, Beverly L Davidson

Mol Ther Nucleic Acids· September 2023

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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, Beverly L Davidson

bioRxiv· August 2023

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Erratum: Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing.
Li Fang, Alex Mas Monteys, Alexandra Dürr, Megan Keiser, Congsheng Cheng, Beverly L Davidson

HGG Adv· July 2023

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Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching.
Jennine M Dawicki-McKenna, Alex J Felix, Elisa A Waxman, Congsheng Cheng, Defne A Amado, Beverly L Davidson

Nat Commun· May 2023

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