Milstein, Stuart

Multivalent N -Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

Nair, J. K., Willoughby, J. L. S., Chan, A., Charisse, K., Alam, M. R., Wang, Q., Hoekstra, M., Kandasamy, P., Kel’in, A. V., Milstein, S., et al. (2014) · Journal of the American Chemical Society

RNA Interference and Gene DeliveryDNA and Nucleic Acid Chemistry

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Therapeutic siRNA silencing in inflammatory monocytes in mice

Leuschner, F., Dutta, P., Gorbatov, R., Novobrantseva, T. I., Donahoe, J. S., Courties, G., Lee, K. M., Kim, J. I., Markmann, J. F., Marinelli, B., et al. (2011) · Nature Biotechnology

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

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Acetyl CoA carboxylase inhibition reduces hepatic steatosis but elevates plasma triglycerides in mice and humans: a bedside to bench investigation

Kim, C.-W., Addy, C., Kusunoki, J., Anderson, N. N., Deja, S., Fu, X., Burgess, S. C., Li, C., Ruddy, M., Chakravarthy, M., et al. (2017) · Cell Metabolism

Liver Disease Diagnosis and TreatmentCancer, Lipids, and Metabolism

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Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates

Foster, D. J., Brown, C. R., Shaikh, S., Trapp, C., Schlegel, M. K., Qian, K., Sehgal, A., Rajeev, K. G., Jadhav, V., Manoharan, M., et al. (2018) · Molecular Therapy

RNA Interference and Gene DeliveryDNA and Nucleic Acid Chemistry

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Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates

Brown, K. M., Nair, J. K., Janas, M. M., Anglero-Rodriguez, Y. I., Dang, L. T. H., Peng, H., Theile, C. S., Castellanos-Rizaldos, E., Brown, C., Foster, D., et al. (2022) · Nature Biotechnology

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

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An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe−/− mice and ameliorates anemia and iron overload in murine β-thalassemia intermedia

Schmidt, P. J., Toudjarska, I., Sendamarai, A. K., Racie, T., Milstein, S., Bettencourt, B. R., Hettinger, J., Bumcrot, D., & Fleming, M. D. (2013) · Blood

Iron Metabolism and DisordersHemoglobinopathies and Related Disorders

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Preclinical development of a subcutaneous ALAS1 RNAi therapeutic for treatment of hepatic porphyrias using circulating RNA quantification

Chan, A., Liebow, A., Yasuda, M., Gan, L., Racie, T., Maier, M., Kuchimanchi, S., Foster, D., Milstein, S., Charisse, K., et al. (2015) · Molecular Therapy — Nucleic Acids

Porphyrin Metabolism and DisordersRNA Interference and Gene Delivery

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Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis

Butler, J. S., Chan, A., Costelha, S., Fishman, S., Willoughby, J. L. S., Borland, T. D., Milstein, S., Foster, D. J., Gonçalves, P., Chen, Q., et al. (2016) · Amyloid

Amyloidosis: Diagnosis, Treatment, OutcomesAlzheimer's disease research and treatments

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