Ellen Sapp

Harvard University — United States

Neuroscience · Life Sciences

47h-index13.2kcitations111works5.52yr avg
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Research Topics

Genetic Neurodegenerative Diseases(75), Mitochondrial Function and Pathology(47), Neurological disorders and treatments(28), Muscle Physiology and Disorders(20), RNA Interference and Gene Delivery(20)

Publications111 total

Localization of mutant huntingtin with HTT Exon1 P90 C-terminal neoepitope antibodies in relation to regional and neuronal vulnerability in forebrain in Q175 mice and human huntington's disease
Journal of Huntington s Disease·2025
Early reduction and impaired targeting of myelin-associated glycoprotein to myelin membranes in Huntington’s disease
2025Open Access
ESC derived human cortical neurons harboring the NACC1 c.892C > T p.R298W missense mutation exhibit molecular differences from controls that influence neuronal maturation
Human Molecular Genetics·2025
Intrastriatal Delivery of a Zinc Finger Protein Targeting the Mutant HTT Gene Allele Obviates Lipid Phenotypes in Brain and Plasma in Huntington's Disease Mice
Human Gene Therapy·2025
Blocking somatic repeat expansion and lowering huntingtin via RNA interference synergize to prevent Huntington’s disease pathogenesis in mice
2025· 3 citedOpen Access
Intrastriatal delivery of a zinc finger protein targeting the mutant HTT gene by AAV9 obviates lipid phenotypes in brain and plasma of zQ175DN HD mice
2025Open Access
Mutant Huntingtin exon1 protein detected in mouse brain with neoepitope antibody: effects of CAG repeat expansion, MSH3 silencing, and aggregation
2025Open Access
Mutant huntingtin exon 1 protein detected in mouse brain with neoepitope antibody: effects of CAG repeat expansion, MutS Homolog 3 silencing and aggregation
Brain Communications·2025· 2 citedOpen Access
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation
Molecular Therapy — Nucleic Acids·2024· 14 citedOpen Access
Enhancing siRNA efficacy in vivo with extended nucleic acid backbones
Nature Biotechnology·2024· 37 cited
mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs <i>In Vivo</i>
Nucleic Acid Therapeutics·2024· 4 citedOpen Access
Chronic pharmacologic manipulation of dopamine transmission ameliorates metabolic disturbance in Trappc9-linked brain developmental syndrome
JCI Insight·2024· 2 citedOpen Access
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation
2024· 1 citedOpen Access
A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system
Nucleic Acids Research·2024· 22 citedOpen Access
mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs <i>in vivo</i>
2024· 2 citedOpen Access
Nacc1 Mutation in Mice Models Rare Neurodevelopmental Disorder with Underlying Synaptic Dysfunction
Journal of Neuroscience·2024· 7 citedOpen Access
Chronic pharmacologic manipulation of dopamine transmission ameliorates metabolic disturbance in syndrome caused by mutated trappc9
2024Open Access
A programmable dual-targeting di-valent siRNA scaffold supports potent multi-gene modulation in the central nervous system
2023· 3 citedOpen Access
Early whole-body mutant huntingtin lowering averts changes in proteins and lipids important for synapse function and white matter maintenance in the LacQ140 mouse model
Neurobiology of Disease·2023· 11 citedOpen Access
Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington’s disease
Molecular Therapy·2023· 7 citedOpen Access
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Frequent Co-authors

Marian DiFiglia(47), Neil Aronin(38), Kimberly B. Kegel(16), Kathryn Chase(12), Anastasia Khvorova(11), Dimas Echeverria(8), Zheng‐Hong Qin(8), Antonio Valencia(8), J. Steven Alexander(8), Reka A. Haraszti(7), Andrew H. Coles(7), Julia F. Alterman(7), Patrick Reeves(7), Marie-Cécile Didiot(6), Matthew Hassler(6), Xueyi Li(6), Nicholas Masso(6), Rachael Miller(5), Bruno M.D.C. Godinho(5), J.-P. G. Vonsattel(4)