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

Molecular therapy. Nucleic acids

Alterman JF, Hall LM, Coles AH, Hassler MR, Didiot MC, Chase K, Abraham J, Sottosanti E, Johnson E, Sapp E, Osborn MF, Difiglia M, Aronin N, Khvorova A.
PMID: 26623938
Mol Ther Nucleic Acids. 2015 Dec 01;4:e266. doi: 10.1038/mtna.2015.38.

Applications of RNA interference for neuroscience research have been limited by a lack of simple and efficient methods to deliver oligonucleotides to primary neurons in culture and to the brain. Here, we show that primary neurons rapidly internalize hydrophobically...

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Alterman JF, Hall LM, Coles AH, et al. Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain. Mol Ther Nucleic Acids. 2015;4:e266doi: 10.1038/mtna.2015.38.
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., Osborn, M. F., Difiglia, M., Aronin, N., & Khvorova, A. (2015). Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain. Molecular therapy. Nucleic acids, 4e266. https://doi.org/10.1038/mtna.2015.38
Alterman, Julia F, et al. "Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain." Molecular therapy. Nucleic acids vol. 4 (2015): e266. doi: https://doi.org/10.1038/mtna.2015.38
Alterman JF, Hall LM, Coles AH, Hassler MR, Didiot MC, Chase K, Abraham J, Sottosanti E, Johnson E, Sapp E, Osborn MF, Difiglia M, Aronin N, Khvorova A. Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain. Mol Ther Nucleic Acids. 2015 Dec 01;4:e266. doi: 10.1038/mtna.2015.38. PMID: 26623938; PMCID: PMC5014532.
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Thermally stable coexistence of liquid and solid phases in gallium nanoparticles.

Nature materials

Losurdo M, Suvorova A, Rubanov S, Hingerl K, Brown AS.
PMID: 27454047
Nat Mater. 2016 Sep;15(9):995-1002. doi: 10.1038/nmat4705. Epub 2016 Jul 25.

Gallium (Ga), a group III metal, is of fundamental interest due to its polymorphism and unusual phase transition behaviours. New solid phases have been observed when Ga is confined at the nanoscale. Herein, we demonstrate the stable coexistence, from...

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Losurdo M, Suvorova A, Rubanov S, et al. Thermally stable coexistence of liquid and solid phases in gallium nanoparticles. Nat Mater. 2016;15(9):995-1002doi: 10.1038/nmat4705.
Losurdo, M., Suvorova, A., Rubanov, S., Hingerl, K., & Brown, A. S. (2016). Thermally stable coexistence of liquid and solid phases in gallium nanoparticles. Nature materials, 15(9), 995-1002. https://doi.org/10.1038/nmat4705
Losurdo, Maria, et al. "Thermally stable coexistence of liquid and solid phases in gallium nanoparticles." Nature materials vol. 15,9 (2016): 995-1002. doi: https://doi.org/10.1038/nmat4705
Losurdo M, Suvorova A, Rubanov S, Hingerl K, Brown AS. Thermally stable coexistence of liquid and solid phases in gallium nanoparticles. Nat Mater. 2016 Sep;15(9):995-1002. doi: 10.1038/nmat4705. Epub 2016 Jul 25. PMID: 27454047.
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Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain.

Journal of drug delivery science and technology

Sanchez-Ramos J, Song S, Kong X, Foroutan P, Martinez G, Dominguez-Viqueria W, Mohapatra S, Mohapatra S, Haraszti RA, Khvorova A, Aronin N, Sava V.
PMID: 29805475
J Drug Deliv Sci Technol. 2018 Feb;43:453-460. doi: 10.1016/j.jddst.2017.11.013. Epub 2017 Nov 21.

The overall objective of the present research was to develop a nanocarrier system for non-invasive delivery to brain of molecules useful for gene therapy. Manganese-containing nanoparticles (mNPs) carrying

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Sanchez-Ramos J, Song S, Kong X, et al. Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain. J Drug Deliv Sci Technol. 2017;43:453-460doi: 10.1016/j.jddst.2017.11.013.
Sanchez-Ramos, J., Song, S., Kong, X., Foroutan, P., Martinez, G., Dominguez-Viqueria, W., Mohapatra, S., Mohapatra, S., Haraszti, R. A., Khvorova, A., Aronin, N., & Sava, V. (2018). Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain. Journal of drug delivery science and technology, 43453-460. https://doi.org/10.1016/j.jddst.2017.11.013
Sanchez-Ramos, Juan, et al. "Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain." Journal of drug delivery science and technology vol. 43 (2018): 453-460. doi: https://doi.org/10.1016/j.jddst.2017.11.013
Sanchez-Ramos J, Song S, Kong X, Foroutan P, Martinez G, Dominguez-Viqueria W, Mohapatra S, Mohapatra S, Haraszti RA, Khvorova A, Aronin N, Sava V. Chitosan-Mangafodipir nanoparticles designed for intranasal delivery of siRNA and DNA to brain. J Drug Deliv Sci Technol. 2018 Feb;43:453-460. doi: 10.1016/j.jddst.2017.11.013. Epub 2017 Nov 21. PMID: 29805475; PMCID: PMC5967853.
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A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons .

Bio-protocol

Alterman JF, Coles AH, Hall LM, Aronin N, Khvorova A, Didiot MC.
PMID: 28966945
Bio Protoc. 2017 Aug 20;7(16). doi: 10.21769/BioProtoc.2501.

Primary neurons represent an ideal cellular system for the identification of therapeutic oligonucleotides for the treatment of neurodegenerative diseases. However, due to the sensitive nature of primary cells, the transfection of small interfering RNAs (siRNA) using classical methods is...

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Alterman JF, Coles AH, Hall LM, et al. A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons . Bio Protoc. 2017;7(16)doi: 10.21769/BioProtoc.2501.
Alterman, J. F., Coles, A. H., Hall, L. M., Aronin, N., Khvorova, A., & Didiot, M. C. (2017). A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons . Bio-protocol, 7(16), . https://doi.org/10.21769/BioProtoc.2501
Alterman, Julia F, et al. "A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons ." Bio-protocol vol. 7,16 (2017). doi: https://doi.org/10.21769/BioProtoc.2501
Alterman JF, Coles AH, Hall LM, Aronin N, Khvorova A, Didiot MC. A High-throughput Assay for mRNA Silencing in Primary Cortical Neurons . Bio Protoc. 2017 Aug 20;7(16). doi: 10.21769/BioProtoc.2501. PMID: 28966945; PMCID: PMC5621760.
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Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep.

JCI insight

Ferguson CM, Godinho BM, Alterman JF, Coles AH, Hassler M, Echeverria D, Gilbert JW, Knox EG, Caiazzi J, Haraszti RA, King RM, Taghian T, Puri A, Moser RP, Gounis MJ, Aronin N, Gray-Edwards H, Khvorova A.
PMID: 34935646
JCI Insight. 2021 Dec 22;6(24). doi: 10.1172/jci.insight.152203.

siRNAs comprise a class of drugs that can be programmed to silence any target gene. Chemical engineering efforts resulted in development of divalent siRNAs (di-siRNAs), which support robust and long-term efficacy in rodent and nonhuman primate brains upon direct...

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Ferguson CM, Godinho BM, Alterman JF, et al. Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep. JCI Insight. 2021;6(24)doi: 10.1172/jci.insight.152203.
Ferguson, C. M., Godinho, B. M., Alterman, J. F., Coles, A. H., Hassler, M., Echeverria, D., Gilbert, J. W., Knox, E. G., Caiazzi, J., Haraszti, R. A., King, R. M., Taghian, T., Puri, A., Moser, R. P., Gounis, M. J., Aronin, N., Gray-Edwards, H., & Khvorova, A. (2021). Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep. JCI insight, 6(24), . https://doi.org/10.1172/jci.insight.152203
Ferguson, Chantal M, et al. "Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep." JCI insight vol. 6,24 (2021). doi: https://doi.org/10.1172/jci.insight.152203
Ferguson CM, Godinho BM, Alterman JF, Coles AH, Hassler M, Echeverria D, Gilbert JW, Knox EG, Caiazzi J, Haraszti RA, King RM, Taghian T, Puri A, Moser RP, Gounis MJ, Aronin N, Gray-Edwards H, Khvorova A. Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep. JCI Insight. 2021 Dec 22;6(24). doi: 10.1172/jci.insight.152203. PMID: 34935646.
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Differential protein expression of mitogen-activated protein kinases in erythrocytes and liver of lamprey Lampetra fluviatilis on the course of prespawning starvation.

Comparative biochemistry and physiology. Part A, Molecular & integrative physiology

Khvorova IA, Nadei OV, Agalakova NI.
PMID: 34728403
Comp Biochem Physiol A Mol Integr Physiol. 2022 Feb;264:111108. doi: 10.1016/j.cbpa.2021.111108. Epub 2021 Oct 30.

The study was designed to identify the types of mitogen-activated protein kinases (MAPKs) in erythrocytes and liver tissues of river lamprey Lampetra fluviatilis and monitor the changes in protein expression levels of found enzymes on the course of prespawning...

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Khvorova IA, Nadei OV, Agalakova NI. Differential protein expression of mitogen-activated protein kinases in erythrocytes and liver of lamprey Lampetra fluviatilis on the course of prespawning starvation. Comp Biochem Physiol A Mol Integr Physiol. 2021;264:111108doi: 10.1016/j.cbpa.2021.111108.
Khvorova, I. A., Nadei, O. V., & Agalakova, N. I. (2022). Differential protein expression of mitogen-activated protein kinases in erythrocytes and liver of lamprey Lampetra fluviatilis on the course of prespawning starvation. Comparative biochemistry and physiology. Part A, Molecular & integrative physiology, 264111108. https://doi.org/10.1016/j.cbpa.2021.111108
Khvorova, Irina A, et al. "Differential protein expression of mitogen-activated protein kinases in erythrocytes and liver of lamprey Lampetra fluviatilis on the course of prespawning starvation." Comparative biochemistry and physiology. Part A, Molecular & integrative physiology vol. 264 (2022): 111108. doi: https://doi.org/10.1016/j.cbpa.2021.111108
Khvorova IA, Nadei OV, Agalakova NI. Differential protein expression of mitogen-activated protein kinases in erythrocytes and liver of lamprey Lampetra fluviatilis on the course of prespawning starvation. Comp Biochem Physiol A Mol Integr Physiol. 2022 Feb;264:111108. doi: 10.1016/j.cbpa.2021.111108. Epub 2021 Oct 30. PMID: 34728403.
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Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep.

JCI insight

Ferguson CM, Godinho BM, Alterman JF, Coles AH, Hassler M, Echeverria D, Gilbert JW, Knox EG, Caiazzi J, Haraszti RA, King RM, Taghian T, Puri A, Moser RP, Gounis MJ, Aronin N, Gray-Edwards H, Khvorova A.
PMID: 34935646
JCI Insight. 2021 Dec 22;6(24). doi: 10.1172/jci.insight.152203.

siRNAs comprise a class of drugs that can be programmed to silence any target gene. Chemical engineering efforts resulted in development of divalent siRNAs (di-siRNAs), which support robust and long-term efficacy in rodent and nonhuman primate brains upon direct...

Cite
Ferguson CM, Godinho BM, Alterman JF, et al. Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep. JCI Insight. 2021;6(24)doi: 10.1172/jci.insight.152203.
Ferguson, C. M., Godinho, B. M., Alterman, J. F., Coles, A. H., Hassler, M., Echeverria, D., Gilbert, J. W., Knox, E. G., Caiazzi, J., Haraszti, R. A., King, R. M., Taghian, T., Puri, A., Moser, R. P., Gounis, M. J., Aronin, N., Gray-Edwards, H., & Khvorova, A. (2021). Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep. JCI insight, 6(24), . https://doi.org/10.1172/jci.insight.152203
Ferguson, Chantal M, et al. "Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep." JCI insight vol. 6,24 (2021). doi: https://doi.org/10.1172/jci.insight.152203
Ferguson CM, Godinho BM, Alterman JF, Coles AH, Hassler M, Echeverria D, Gilbert JW, Knox EG, Caiazzi J, Haraszti RA, King RM, Taghian T, Puri A, Moser RP, Gounis MJ, Aronin N, Gray-Edwards H, Khvorova A. Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep. JCI Insight. 2021 Dec 22;6(24). doi: 10.1172/jci.insight.152203. PMID: 34935646.
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Ultrathin High-Quality SnTe Nanoplates for Fabricating Flexible Near-Infrared Photodetectors.

ACS applied materials & interfaces

Liu J, Li X, Wang H, Yuan G, Suvorova A, Gain S, Ren Y, Lei W.
PMID: 32585086
ACS Appl Mater Interfaces. 2020 Jul 15;12(28):31810-31822. doi: 10.1021/acsami.0c07847. Epub 2020 Jul 06.

This work demonstrates a controlled van der Waals growth of two-dimensional SnTe nanoplates on mica substrates and their applications in flexible near-infrared photodetectors. The growth of nonlayered rock-salt structured SnTe crystals into two-dimensional SnTe nanoplate structures is mainly caused...

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Liu J, Li X, Wang H, et al. Ultrathin High-Quality SnTe Nanoplates for Fabricating Flexible Near-Infrared Photodetectors. ACS Appl Mater Interfaces. 2020;12(28):31810-31822doi: 10.1021/acsami.0c07847.
Liu, J., Li, X., Wang, H., Yuan, G., Suvorova, A., Gain, S., Ren, Y., & Lei, W. (2020). Ultrathin High-Quality SnTe Nanoplates for Fabricating Flexible Near-Infrared Photodetectors. ACS applied materials & interfaces, 12(28), 31810-31822. https://doi.org/10.1021/acsami.0c07847
Liu, Junliang, et al. "Ultrathin High-Quality SnTe Nanoplates for Fabricating Flexible Near-Infrared Photodetectors." ACS applied materials & interfaces vol. 12,28 (2020): 31810-31822. doi: https://doi.org/10.1021/acsami.0c07847
Liu J, Li X, Wang H, Yuan G, Suvorova A, Gain S, Ren Y, Lei W. Ultrathin High-Quality SnTe Nanoplates for Fabricating Flexible Near-Infrared Photodetectors. ACS Appl Mater Interfaces. 2020 Jul 15;12(28):31810-31822. doi: 10.1021/acsami.0c07847. Epub 2020 Jul 06. PMID: 32585086.
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Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition.

iScience

Haraszti RA, Miller R, Dubuke ML, Rockwell HE, Coles AH, Sapp E, Didiot MC, Echeverria D, Stoppato M, Sere YY, Leszyk J, Alterman JF, Godinho BMDC, Hassler MR, McDaniel J, Narain NR, Wollacott R, Wang Y, Shaffer SA, Kiebish MA, DiFiglia M, Aronin N, Khvorova A.
PMID: 31195240
iScience. 2019 Jun 28;16:230-241. doi: 10.1016/j.isci.2019.05.029. Epub 2019 May 27.

Exosomes can serve as delivery vehicles for advanced therapeutics. The components necessary and sufficient to support exosomal delivery have not been established. Here we connect biochemical composition and activity of exosomes to optimize exosome-mediated delivery of small interfering RNAs...

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Haraszti RA, Miller R, Dubuke ML, et al. Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition. iScience. 2019;16:230-241doi: 10.1016/j.isci.2019.05.029.
Haraszti, R. A., Miller, R., Dubuke, M. L., Rockwell, H. E., Coles, A. H., Sapp, E., Didiot, M. C., Echeverria, D., Stoppato, M., Sere, Y. Y., Leszyk, J., Alterman, J. F., Godinho, B. M. D. C., Hassler, M. R., McDaniel, J., Narain, N. R., Wollacott, R., Wang, Y., Shaffer, S. A., Kiebish, M. A., DiFiglia, M., Aronin, N., & Khvorova, A. (2019). Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition. iScience, 16230-241. https://doi.org/10.1016/j.isci.2019.05.029
Haraszti, Reka Agnes, et al. "Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition." iScience vol. 16 (2019): 230-241. doi: https://doi.org/10.1016/j.isci.2019.05.029
Haraszti RA, Miller R, Dubuke ML, Rockwell HE, Coles AH, Sapp E, Didiot MC, Echeverria D, Stoppato M, Sere YY, Leszyk J, Alterman JF, Godinho BMDC, Hassler MR, McDaniel J, Narain NR, Wollacott R, Wang Y, Shaffer SA, Kiebish MA, DiFiglia M, Aronin N, Khvorova A. Serum Deprivation of Mesenchymal Stem Cells Improves Exosome Activity and Alters Lipid and Protein Composition. iScience. 2019 Jun 28;16:230-241. doi: 10.1016/j.isci.2019.05.029. Epub 2019 May 27. PMID: 31195240; PMCID: PMC6562145.
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Single-Stranded Phosphorothioated Regions Enhance Cellular Uptake of Cholesterol-Conjugated siRNA but Not Silencing Efficacy.

Molecular therapy. Nucleic acids

Ly S, Echeverria D, Sousa J, Khvorova A.
PMID: 32818923
Mol Ther Nucleic Acids. 2020 Sep 04;21:991-1005. doi: 10.1016/j.omtn.2020.07.029. Epub 2020 Jul 25.

Small interfering RNAs (siRNAs) have potential to silence virtually any disease-causing gene but require chemical modifications for delivery to the tissue and cell of interest. Previously, we demonstrated that asymmetric, phosphorothioate (PS)-modified, chemically stabilized, cholesterol-conjugated siRNAs, called hsiRNAs, support...

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Ly S, Echeverria D, Sousa J, et al. Single-Stranded Phosphorothioated Regions Enhance Cellular Uptake of Cholesterol-Conjugated siRNA but Not Silencing Efficacy. Mol Ther Nucleic Acids. 2020;21:991-1005doi: 10.1016/j.omtn.2020.07.029.
Ly, S., Echeverria, D., Sousa, J., & Khvorova, A. (2020). Single-Stranded Phosphorothioated Regions Enhance Cellular Uptake of Cholesterol-Conjugated siRNA but Not Silencing Efficacy. Molecular therapy. Nucleic acids, 21991-1005. https://doi.org/10.1016/j.omtn.2020.07.029
Ly, Socheata, et al. "Single-Stranded Phosphorothioated Regions Enhance Cellular Uptake of Cholesterol-Conjugated siRNA but Not Silencing Efficacy." Molecular therapy. Nucleic acids vol. 21 (2020): 991-1005. doi: https://doi.org/10.1016/j.omtn.2020.07.029
Ly S, Echeverria D, Sousa J, Khvorova A. Single-Stranded Phosphorothioated Regions Enhance Cellular Uptake of Cholesterol-Conjugated siRNA but Not Silencing Efficacy. Mol Ther Nucleic Acids. 2020 Sep 04;21:991-1005. doi: 10.1016/j.omtn.2020.07.029. Epub 2020 Jul 25. PMID: 32818923; PMCID: PMC7452107.
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Designer spin pseudomolecule implemented with trapped ions in a magnetic gradient.

Physical review letters

Khromova A, Piltz Ch, Scharfenberger B, Gloger TF, Johanning M, Varón AF, Wunderlich Ch.
PMID: 23003581
Phys Rev Lett. 2012 Jun 01;108(22):220502. doi: 10.1103/PhysRevLett.108.220502. Epub 2012 Jun 01.

We report on the experimental investigation of an individual pseudomolecule using trapped ions with adjustable magnetically induced J-type coupling between spin states. Resonances of individual spins are well separated and are addressed with high fidelity. Quantum gates are carried...

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Khromova A, Piltz Ch, Scharfenberger B, et al. Designer spin pseudomolecule implemented with trapped ions in a magnetic gradient. Phys Rev Lett. 2012;108(22):220502doi: 10.1103/PhysRevLett.108.220502.
Khromova, A., Piltz, C. h., Scharfenberger, B., Gloger, T. F., Johanning, M., Varón, A. F., & Wunderlich, C. h. (2012). Designer spin pseudomolecule implemented with trapped ions in a magnetic gradient. Physical review letters, 108(22), 220502. https://doi.org/10.1103/PhysRevLett.108.220502
Khromova, A, et al. "Designer spin pseudomolecule implemented with trapped ions in a magnetic gradient." Physical review letters vol. 108,22 (2012): 220502. doi: https://doi.org/10.1103/PhysRevLett.108.220502
Khromova A, Piltz Ch, Scharfenberger B, Gloger TF, Johanning M, Varón AF, Wunderlich Ch. Designer spin pseudomolecule implemented with trapped ions in a magnetic gradient. Phys Rev Lett. 2012 Jun 01;108(22):220502. doi: 10.1103/PhysRevLett.108.220502. Epub 2012 Jun 01. PMID: 23003581.
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A new metal-free carbon hybrid for enhanced photocatalysis.

ACS applied materials & interfaces

Sun H, Zhou G, Wang Y, Suvorova A, Wang S.
PMID: 25212502
ACS Appl Mater Interfaces. 2014 Oct 08;6(19):16745-54. doi: 10.1021/am503820h. Epub 2014 Sep 25.

Carbon nitride (C3N4) is a layered, stable, and polymeric metal-free material that has been discovered as a visible-light-response photocatalyst. Owing to C3N4 having a higher conduction band position, most previous studies have been focused on its reduction capability for...

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Sun H, Zhou G, Wang Y, et al. A new metal-free carbon hybrid for enhanced photocatalysis. ACS Appl Mater Interfaces. 2014;6(19):16745-54doi: 10.1021/am503820h.
Sun, H., Zhou, G., Wang, Y., Suvorova, A., & Wang, S. (2014). A new metal-free carbon hybrid for enhanced photocatalysis. ACS applied materials & interfaces, 6(19), 16745-54. https://doi.org/10.1021/am503820h
Sun, Hongqi, et al. "A new metal-free carbon hybrid for enhanced photocatalysis." ACS applied materials & interfaces vol. 6,19 (2014): 16745-54. doi: https://doi.org/10.1021/am503820h
Sun H, Zhou G, Wang Y, Suvorova A, Wang S. A new metal-free carbon hybrid for enhanced photocatalysis. ACS Appl Mater Interfaces. 2014 Oct 08;6(19):16745-54. doi: 10.1021/am503820h. Epub 2014 Sep 25. PMID: 25212502.
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