Marian DiFiglia

Harvard University — United States

Neuroscience · Life Sciences

76h-index26.8kcitations200works6.22yr avg
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Research Topics

Genetic Neurodegenerative Diseases(110), Mitochondrial Function and Pathology(67), Neurological disorders and treatments(43), Neuroscience and Neuropharmacology Research(31), Muscle Physiology and Disorders(30)

Publications200 total

Early reduction and impaired targeting of myelin-associated glycoprotein to myelin membranes in Huntington’s disease
2025Open Access
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
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
Towards Standardizing Nomenclature in Huntington’s Disease Research
Journal of Huntington s Disease·2024· 7 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
Contributors
Elsevier eBooks·2024
Huntingtin lowering therapeutics
Elsevier eBooks·2024
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
Age-Dependent Increase in Tau Phosphorylation at Serine 396 in Huntington’s Disease Prefrontal Cortex
Journal of Huntington s Disease·2023· 7 citedOpen Access
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Frequent Co-authors

Neil Aronin(30), Ellen Sapp(25), Kathryn Chase(9), Kimberly B. Kegel(9), Anastasia Khvorova(5), Zheng‐Hong Qin(5), J.-P. G. Vonsattel(4), Frederick M. Boyce(4), Reka A. Haraszti(4), Marie-Cécile Didiot(4), Andrew H. Coles(4), Jean‐Paul Vonsattel(3), Manho Kim(3), Charmian McIntyre(3), Julia F. Alterman(3), Matthew Hassler(3), Yun Joong Kim(3), Yumei Wang(3), Pedro Pasik(3), Tauba Pasik(3)