Rajeev, Kallanthottathil G.

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

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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

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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

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

Musunuru, K., Chadwick, A. C., Mizoguchi, T., Garcia, S. P., DeNizio, J. E., Reiss, C. W., Wang, K., Iyer, S., Dutta, C., Clendaniel, V., et al. (2021) · Nature

CRISPR and Genetic EngineeringLipoproteins and Cardiovascular Health

Cited by 793

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

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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

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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

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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 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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