Sapp, Ellen

Huntingtin is a cytoplasmic protein associated with vesicles in human and rat brain neurons

DiFiglia, M., Sapp, E., Chase, K., Schwarz, C., Meloni, A., Young, C., Martin, E., Vonsattel, J.-P., Carraway, R., Reeves, S. A., et al. (1995) · Neuron

Genetic Neurodegenerative DiseasesCellular transport and secretion

Cited by 749

High-resolution proteomic and lipidomic analysis of exosomes and microvesicles from different cell sources

Haraszti, R. A., Didiot, M., Sapp, E., Leszyk, J., Shaffer, S. A., Rockwell, H. E., Gao, F., Narain, N. R., DiFiglia, M., Kiebish, M. A., et al. (2016) · Journal of Extracellular Vesicles

Extracellular vesicles in diseaseNanoparticle-Based Drug Delivery

Cited by 746

Exosomes Produced from 3D Cultures of MSCs by Tangential Flow Filtration Show Higher Yield and Improved Activity

Haraszti, R. A., Miller, R., Stoppato, M., Sere, Y. Y., Coles, A., Didiot, M.-C., Wollacott, R., Sapp, E., Dubuke, M. L., Li, X., et al. (2018) · Molecular Therapy

Extracellular vesicles in diseaseMesenchymal stem cell research

Cited by 567

Huntingtin Expression Stimulates Endosomal–Lysosomal Activity, Endosome Tubulation, and Autophagy

Kegel, K. B., Kim, M., Sapp, E., McIntyre, C., Castaño, J. G., Aronin, N., & DiFiglia, M. (2000) · The Journal of Neuroscience

Genetic Neurodegenerative DiseasesAutophagy in Disease and Therapy

Cited by 533

Exosome-mediated delivery of hydrophobically modified siRNA for huntingtin mRNA silencing

Didiot, M.-C., Hall, L. M., Coles, A. H., Haraszti, R. A., Godinho, B. M., Chase, K., Sapp, E., Ly, S., Alterman, J. F., Hassler, M. R., et al. (2016) · Molecular Therapy

RNA Interference and Gene DeliveryExtracellular vesicles in disease

Cited by 483

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

Alterman, J. F., Godinho, B. M. D. C., Hassler, M. R., Ferguson, C. M., Echeverria, D., Sapp, E., Haraszti, R. A., Coles, A. H., Conroy, F., Miller, R., et al. (2019) · Nature Biotechnology

RNA Interference and Gene DeliveryDNA and Nucleic Acid Chemistry

Cited by 227

Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain

Alterman, J. F., Hall, L. M., Coles, A. H., Hassler, M. R., Didiot, M.-C., Chase, K., Abraham, J., Sottosanti, E., Johnson, E., Sapp, E., et al. (2015) · Molecular Therapy - Nucleic Acids

RNA Interference and Gene DeliveryGenetic Neurodegenerative Diseases

Cited by 158

KEAP1-modifying small molecule reveals muted NRF2 signaling responses in neural stem cells from Huntington's disease patients

Quinti, L., Dayalan Naidu, S., Träger, U., Chen, X., Kegel-Gleason, K., Llères, D., Connolly, C., Chopra, V., Low, C., Moniot, S., et al. (2017) · Proceedings of the National Academy of Sciences

Genomics, phytochemicals, and oxidative stressGenetic Neurodegenerative Diseases

Cited by 140

Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease

Pfister, E. L., DiNardo, N., Mondo, E., Borel, F., Conroy, F., Fraser, C., Gernoux, G., Han, X., Hu, D., Johnson, E., et al. (2018) · Human Gene Therapy

Genetic Neurodegenerative DiseasesRNA Interference and Gene Delivery

Cited by 95

Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin

Didiot, M.-C., Ferguson, C. M., Ly, S., Coles, A. H., Smith, A. O., Bicknell, A. A., Hall, L. M., Sapp, E., Echeverria, D., Pai, A. A., et al. (2018) · Cell Reports

Genetic Neurodegenerative DiseasesRNA Interference and Gene Delivery

Cited by 64

Glucose transporter 3 is a rab11-dependent trafficking cargo and its transport to the cell surface is reduced in neurons of CAG140 Huntington’s disease mice

McClory, H., Williams, D., Sapp, E., Gatune, L. W., Wang, P., DiFiglia, M., & Li, X. (2014) · Acta Neuropathologica Communications

Genetic Neurodegenerative DiseasesCellular transport and secretion

Cited by 60

Synthesis and Evaluation of Parenchymal Retention and Efficacy of a Metabolically Stable O-Phosphocholine-N-docosahexaenoyl-l-serine siRNA Conjugate in Mouse Brain

Nikan, M., Osborn, M. F., Coles, A. H., Biscans, A., Godinho, B. M. D. C., Haraszti, R. A., Sapp, E., Echeverria, D., DiFiglia, M., Aronin, N., et al. (2017) · Bioconjugate Chemistry

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 35