DiFiglia, Marian

Guidelines for the use and interpretation of assays for monitoring autophagy in higher eukaryotes

Klionsky, D. J., Abeliovich, H., Agostinis, P., Agrawal, D. K., Aliev, G., Askew, D. S., Baba, M., Baehrecke, E. H., Bahr, B. A., Ballabio, A., et al. (2008) · Autophagy

Autophagy in Disease and TherapyCellular transport and secretion

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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

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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

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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

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Allele-selective lowering of mutant HTT protein by HTT–LC3 linker compounds

Li, Z., Wang, C., Wang, Z., Zhu, C., Li, J., Sha, T., Ma, L., Gao, C., Yang, Y., Sun, Y., et al. (2019) · Nature

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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

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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

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Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients

Pfister, E. L., Kennington, L., Straubhaar, J., Wagh, S., Liu, W., DiFiglia, M., Landwehrmeyer, B., Vonsattel, J.-P., Zamore, P. D., & Aronin, N. (2009) · Current Biology

Genetic Neurodegenerative DiseasesRNA Interference and Gene Delivery

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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

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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

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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

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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

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The mTOR kinase inhibitor Everolimus decreases S6 kinase phosphorylation but fails to reduce mutant huntingtin levels in brain and is not neuroprotective in the R6/2 mouse model of Huntington's disease

Fox, J. H., Connor, T., Chopra, V., Dorsey, K., Kama, J. A., Bleckmann, D., Betschart, C., Hoyer, D., Frentzel, S., DiFiglia, M., et al. (2010) · Molecular Neurodegeneration

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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

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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

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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

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Rapamycin prevents the mutant huntingtin-suppressed GLT-1 expression in cultured astrocytes

Chen, L.-L., Wu, J.-C., Wang, L.-H., Wang, J., Qin, Z.-H., Difiglia, M., & Lin, F. (2012) · Acta Pharmacologica Sinica

Genetic Neurodegenerative DiseasesAutophagy in Disease and Therapy

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