Manoharan, Muthiah

Silencing of microRNAs in vivo with ‘antagomirs’

Krützfeldt, J., Rajewsky, N., Braich, R., Rajeev, K. G., Tuschl, T., Manoharan, M., & Stoffel, M. (2005) · Nature

MicroRNA in disease regulationRNA Interference and Gene Delivery

Cited by 3,949

Rational design of cationic lipids for siRNA delivery

Semple, S. C., Akinc, A., Chen, J., Sandhu, A. P., Mui, B. L., Cho, C. K., Sah, D. W. Y., Stebbing, D., Crosley, E. J., Yaworski, E., et al. (2010) · Nature Biotechnology

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 1,822

The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

Akinc, A., Maier, M. A., Manoharan, M., Fitzgerald, K., Jayaraman, M., Barros, S., Ansell, S., Du, X., Hope, M. J., Madden, T. D., et al. (2019) · Nature Nanotechnology

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 1,636

RNAi-mediated gene silencing in non-human primates

Zimmermann, T. S., Lee, A. C. H., Akinc, A., Bramlage, B., Bumcrot, D., Fedoruk, M. N., Harborth, J., Heyes, J. A., Jeffs, L. B., John, M., et al. (2006) · Nature

RNA Interference and Gene DeliveryVirus-based gene therapy research

Cited by 1,357

Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo

Jayaraman, M., Ansell, S. M., Mui, B. L., Tam, Y. K., Chen, J., Du, X., Butler, D., Eltepu, L., Matsuda, S., Narayanannair, J. K., et al. (2012) · Angewandte Chemie International Edition

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 1,267

Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms

Akinc, A., Querbes, W., De, S., Qin, J., Frank-Kamenetsky, M., Jayaprakash, K. N., Jayaraman, M., Rajeev, K. G., Cantley, W. L., Dorkin, J. R., et al. (2010) · Molecular Therapy

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 1,261

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

Cited by 1,141

Mechanisms and optimization of in vivo delivery of lipophilic siRNAs

Wolfrum, C., Shi, S., Jayaprakash, K. N., Jayaraman, M., Wang, G., Pandey, R. K., Rajeev, K. G., Nakayama, T., Charrise, K., Ndungo, E. M., et al. (2007) · Nature Biotechnology

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 974

Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown

Wittrup, A., Ai, A., Liu, X., Hamar, P., Trifonova, R., Charisse, K., Manoharan, M., Kirchhausen, T., & Lieberman, J. (2015) · Nature Biotechnology

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 693

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

Maier, M. A., Jayaraman, M., Matsuda, S., Liu, J., Barros, S., Querbes, W., Tam, Y. K., Ansell, S. M., Kumar, V., Qin, J., et al. (2013) · Molecular Therapy

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 625

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

Cited by 325

Investigating the pharmacodynamic durability of GalNAc–siRNA conjugates

Brown, C. R., Gupta, S., Qin, J., Racie, T., He, G., Lentini, S., Malone, R., Yu, M., Matsuda, S., Shulga-Morskaya, S., et al. (2020) · Nucleic Acids Research

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 285

Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity

Janas, M. M., Schlegel, M. K., Harbison, C. E., Yilmaz, V. O., Jiang, Y., Parmar, R., Zlatev, I., Castoreno, A., Xu, H., Shulga-Morskaya, S., et al. (2018) · Nature Communications

RNA Interference and Gene DeliveryDrug-Induced Hepatotoxicity and Protection

Cited by 279

Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates

Nair, J. K., Attarwala, H., Sehgal, A., Wang, Q., Aluri, K., Zhang, X., Gao, M., Liu, J., Indrakanti, R., Schofield, S., et al. (2017) · Nucleic Acids Research

RNA Interference and Gene DeliveryDNA and Nucleic Acid Chemistry

Cited by 278

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

Cited by 258

Lipid Nanoparticle Delivery of siRNA to Silence Neuronal Gene Expression in the Brain

Rungta, R. L., Choi, H. B., Lin, P. J., Ko, R. W., Ashby, D., Nair, J., Manoharan, M., Cullis, P. R., & MacVicar, B. A. (2013) · Molecular Therapy—Nucleic Acids

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 165

Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo**

Jayaraman, M., Ansell, S. M., Mui, B. L., Tam, Y. K., Chen, J., Du, X., Butler, D., Eltepu, L., Matsuda, S., Narayanannair, J. K., et al. (2012) · Angewandte Chemie

RNA Interference and Gene DeliveryNanoparticle-Based Drug Delivery

Cited by 150

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

Cited by 128

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

Cited by 113

The RNA-binding protein vigilin regulates VLDL secretion through modulation of Apob mRNA translation

Mobin, M. B., Gerstberger, S., Teupser, D., Campana, B., Charisse, K., Heim, M. H., Manoharan, M., Tuschl, T., & Stoffel, M. (2016) · Nature Communications

RNA Research and SplicingCholesterol and Lipid Metabolism

Cited by 55