Cytosine base editing inhibits hepatitis B virus replication and reduces HBsAg expression in vitro and in vivo

Authors: Smekalova, Elena M., Martinez, Maria G., Combe, Emmanuel, Kumar, Anuj, Dejene, Selam, Leboeuf, Dominique, Chen, Chao-Ying, Dorkin, J. Robert, Shuang, Lan Shuan, Kieft, Sarah, Young, Lauren, Barrera, Luis Alberto, Packer, Michael S., Ciaramella, Giuseppe, Testoni, Barbara, Gregoire, Francine, Zoulim, Fabien

Source: In Molecular Therapy - Nucleic Acids 12 March 2024 35(1)

Rapid, Single-Cell Analysis and Discovery of Vectored mRNA Transfection In Vivo with a loxP-Flanked tdTomato Reporter Mouse

Authors: Kauffman, Kevin J., Oberli, Matthias A., Dorkin, J. Robert, Hurtado, Juan E., Kaczmarek, James C., Bhadani, Shivani, Wyckoff, Jeff, Langer, Robert, Jaklenec, Ana, Anderson, Daniel G.

Source: In Molecular Therapy - Nucleic Acids 2 March 2018 10:55-63

Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates

Authors: Dong, Yizhou, Love, Kevin T., Dorkin, J. Robert, Sirirungruang, Sasilada, Zhang, Yunlong, Chen, Delai, Bogorad, Roman L., Yin, Hao, Chen, Yi, Vegas, Arturo J., Alabi, Christopher A., Sahay, Gaurav, Olejnik, Karsten T., Wang, Weiheng, Schroeder, Avi, Lytton-Jean, Abigail K. R., Siegwart, Daniel J., Akinc, Akin, Barnes, Carmen, Barros, Scott A., Carioto, Mary, Fitzgerald, Kevin, Hettinger, Julia, Kumar, Varun, Novobrantseva, Tatiana I., Qin, June, Querbes, William, Koteliansky, Victor, Langer, Robert, Anderson, Daniel G.

Source: Proceedings of the National Academy of Sciences of the United States of America, 2014 Mar . 111(11), 3955-3960.

Access URL: https://www.jstor.org/stable/23770965

Lipid-Like Materials for Low-Dose, in vivo Gene Silencing

Authors: Love, Kevin T., Mahon, Kerry P., Levins, Christopher G., Whitehead, Kathryn A., Querbes, William, Dorkin, J. Robert, Qin, June, Cantley, William, Qin, Liu Liang, Racie, Timothy, Frank-Kamenetsky, Maria, Yip, Ka Ning, Alvarez, Rene, Sah, Dinah W. Y., de Fougerolles, Antonin, Fitzgerald, Kevin, Koteliansky, Victor, Akinc, Akin, Langer, Robert, Anderson, Daniel G., Klibanov, Alexander

Source: Proceedings of the National Academy of Sciences of the United States of America, 2010 Feb 01. 107(5), 1864-1869.

Access URL: https://www.jstor.org/stable/40536500

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

Authors: Akinc, Akin, Querbes, William, De, Soma, Qin, June, Frank-Kamenetsky, Maria, Jayaprakash, K. Narayanannair, Jayaraman, Muthusamy, Rajeev, Kallanthottathil G, Cantley, William L, Dorkin, J Robert, Butler, James S, Qin, LiuLiang, Racie, Timothy, Sprague, Andrew, Fava, Eugenio, Zeigerer, Anja, Hope, Michael J, Zerial, Marino, Sah, Dinah WY, Fitzgerald, Kevin, Tracy, Mark A, Manoharan, Muthiah, Koteliansky, Victor, Fougerolles, Antonin de, Maier, Martin A

Source: In Molecular Therapy July 2010 18(7):1357-1364

Assessing siRNA Pharmacodynamics in a Luciferase-expressing Mouse

Authors: Svensson, Robert U, Shey, Michael R, Ballas, Zuhair K, Dorkin, J Robert, Goldberg, Michael, Akinc, Akin, Langer, Robert, Anderson, Daniel G, Bumcrot, David, Henry, Michael D

Source: In Molecular Therapy December 2008 16(12):1995-2001

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Poly(glycoamidoamine) Brushes Formulated Nanomaterials for Systemic siRNA and mRNA Delivery in Vivo.

Authors: Yizhou Dong, Dorkin, J. Robert, Weiheng Wang, Chang, Philip H., Webber, Matthew J., Tang, Benjamin C., Junghoon Yang, Abutbul-Ionita, Inbal, Danino, Dganit, DeRosa, Frank, Heartlein, Michael, Langer, Robert, Anderson, Daniel G.

Source: Nano Letters; Feb2016, Vol. 16 Issue 2, p842-848, 7p

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Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates.

Authors: Yizhou Dong, Love, Kevin T., Dorkin, J. Robert, Sirirungruang, Sasilada, Yunlong Zhang, Delai Chen, Bogorad, Roman L., Hao Yin, Yi Chen, Vegas, Arturo J., Alabi, Christopher A., Sahay, Gaurav, Olejnik, Karsten T., Weiheng Wang, Schroeder, Avi, Lytton-Jean, Abigail K. R., Siegwart, Daniel J., Akinc, Akin, Barnes, Carmen, Barros, Scott A.

Source: Proceedings of the National Academy of Sciences of the United States of America; 3/18/2014, Vol. 111 Issue 11, p3955-3960, 6p

Subject Terms: SMALL interfering RNA, NANOPARTICLES, GENETIC disorder treatment, LIPOPROTEINS, LABORATORY mice, APOLIPOPROTEIN E, ENDOTHELIAL cells, THERAPEUTICS

In Vitro–In VivoTranslation of Lipid Nanoparticles for Hepatocellular siRNA Delivery

Authors: Whitehead, Kathryn A., Matthews, Jonathan, Chang, Philip H., Niroui, Farnaz, Dorkin, J. Robert, Severgnini, Mariano, Anderson, Daniel G.

Source: ACS Nano; August 2012, Vol. 6 Issue: 8 p6922-6929, 8p