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Ellen Sapp

Harvard University

74

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Promise and challenges of dystonia brain banking: establishing a human tissue repository for studies of X-Linked Dystonia-Parkinsonism.

2021 Journal of Neural Transmission
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Allele-specific knockdown of mutant HTT protein via editing at coding region SNP heterozygosities.

2021 Human Gene Therapy
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Targeting Tau Mitigates Mitochondrial Fragmentation and Oxidative Stress in Amyotrophic Lateral Sclerosis

2021 Molecular Neurobiology
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Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity.

2021 Nucleic Acids Research
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Protein changes in synaptosomes of Huntington's disease knock-in mice are dependent on age and brain region.

2020 Neurobiology of Disease
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Assessing average somatic CAG repeat instability at the protein level

2019 Scientific Reports
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Huntingtin associates with the actin cytoskeleton and α-actinin isoforms to influence stimulus dependent morphology changes

2019 PLOS ONE
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A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

2019 Nature Biotechnology
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Rac1 Activity Is Modulated by Huntingtin and Dysregulated in Models of Huntington’s Disease

2019 Journal of Huntington's disease
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Serum deprivation of mesenchymal stem cells improves exosome activity and alters lipid and protein composition

2019 iScience
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