Development of an mRNA replacement therapy for phenylketonuria von Perez-Garcia, Carlos G., Diaz-Trelles, Ramon, Vega, Jerel Boyd, Bao, Yanjie, Sablad, Marciano, Limphong, Patty, Chikamatsu, Simon, Yu, Hailong, Taylor, Wendy, Karmali, Priya P., Tachikawa, Kiyoshi, Chivukula, Padmanabh

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Effect of PEGylation on Biodistribution and Gene Silencing of siRNA/Lipid Nanoparticle Complexes von Bao, Yanjie, Jin, Yi, Chivukula, Padmanabh, Zhang, Jun, Liu, Yun, Liu, Jian, Clamme, Jean-Pierre, Mahato, Ram I., Ng, Dominic, Ying, Wenbin, Wang, Yiting, Yu, Lei

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Preclinical evaluation of a fully human, quadrivalent-hantavirus polyclonal antibody derived from a non-human source von Brocato, Rebecca L, Wu, Hua, Kwilas, Steven A, Principe, Lucia M, Josleyn, Matthew, Shamblin, Joshua, Chivukula, Padmanabh, Bausch, Christoph, Luke, Thomas, Sullivan, Eddie J, Hooper, Jay W

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Anti-HFRS Human IgG Produced in Transchromosomic Bovines Has Potent Hantavirus Neutralizing Activity and Is Protective in Animal Models von Perley, Casey C., Brocato, Rebecca L., Wu, Hua, Bausch, Christoph, Karmali, Priya P., Vega, Jerel B., Cohen, Melanie V., Somerville, Brandon, Kwilas, Steven A., Principe, Lucia M., Shamblin, Joshua, Chivukula, Padmanabh, Sullivan, Eddie, Hooper, Jay W.

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Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS von Hirunagi, Tomoki, Sahashi, Kentaro, Tachikawa, Kiyoshi, Leu, Angel I., Nguyen, Michelle, Mukthavaram, Rajesh, Karmali, Priya P., Chivukula, Padmanabh, Tohnai, Genki, Iida, Madoka, Onodera, Kazunari, Ohyama, Manabu, Okada, Yohei, Okano, Hideyuki, Katsuno, Masahisa

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Restoring ornithine transcarbamylase (OTC) activity in an OTC‐deficient mouse model using LUNAR‐OTC mRNA von Yu, Hailong, Brewer, Edward, Shields, Michael, Crowder, Michael, Sacchetti, Cristiano, Soontornniyomkij, Benchawanna, Dou, Dandan, Clemente, Brenda, Sablad, Marciano, Chivukula, Padmanabh, Hughes, Steve, Roberts, Scott, Rajappan, Kumar, Tannis, Steve, Sekulovich, Rose, Parker, Suezanne, Limphong, Pattraranee

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Lipid nanoparticle delivers phenylalanine ammonia lyase mRNA to the liver leading to catabolism and clearance of phenylalanine in a phenylketonuria mouse model von Diaz-Trelles, Ramon, Lee, Sharon, Kuakini, Kristen, Park, Jenny, Dukanovic, Adrian, Gonzalez, Jose A., Dam, Thanhchau, Kim, Jae Heon, Vega, Jerel Boyd, Sablad, Marciano, Karmali, Priya P., Tachikawa, Kiyoshi, Chivukula, Padmanabh

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Synthesis and characterization of novel aromatic azo bond-containing pH-sensitive and hydrolytically cleavable IPN hydrogels von Chivukula, Padmanabh, Dušek, Karel, Wang, Dong, Dušková-Smrčková, Mirka, Kopečková, Pavla, Kopeček, Jindřich

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Property-Driven Design and Development of Lipids for Efficient Delivery of siRNA von Rajappan, Kumar, Tanis, Steven P, Mukthavaram, Rajesh, Roberts, Scott, Nguyen, Michelle, Tachikawa, Kiyoshi, Sagi, Amit, Sablad, Marciano, Limphong, Pattraranee, Leu, Angel, Yu, Hailong, Chivukula, Padmanabh, Payne, Joseph E, Karmali, Priya

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Synthesis and bioactivity of readily hydrolysable novel cationic lipids for potential lung delivery application of mRNAs von Pei, Yihua, Bao, Yanjie, Sacchetti, Cristiano, Brady, Juthamart, Gillard, Kyra, Yu, Hailong, Roberts, Scott, Rajappan, Kumar, Tanis, Steven P., Perez-Garcia, Carlos G., Chivukula, Padmanabh, Karmali, Priya P.

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Development of a Safe and Scalable Process for the Production of a High-Purity Thiocarbamate-Based Ionizable Lipid as an Excipient in mRNA-Encapsulating Lipid Nanoparticles von Rajappan, Kumar, Tanis, Steven P, Roberts, Scott, Karmali, Priya, Chivukula, Padmanabh, Zhuang, Jin, Xin, Mingxing, Zheng, Manman, Edmonds, Ian

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Novel biodegradable lipid nano complex for siRNA delivery significantly improving the chemosensitivity of human colon cancer stem cells to paclitaxel von Liu, Changxing, Zhao, Gang, Liu, Jian, Ma, Nianchun, Chivukula, Padmanabh, Perelman, Loren, Okada, Keisaku, Chen, Zongyou, Gough, David, Yu, Lei

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Immune modulatory nanoparticle therapeutics for intracerebral glioma von Yaghi, Nasser K, Wei, Jun, Hashimoto, Yuuri, Kong, Ling-Yuan, Gabrusiewicz, Konrad, Nduom, Edjah K, Ling, Xiaoyang, Huang, Neal, Zhou, Shouhao, Kerrigan, Brittany C Parker, Levine, Jonathan M, Fajt, Virginia R, Levine, Gwendolyn, Porter, Brian F, Marcusson, Eric G, Tachikawa, Kiyoshi, Chivukula, Padmanabh, Webb, David C, Payne, Joseph E, Heimberger, Amy B

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CD8 + T cells mediate protection against Zika virus induced by an NS3-based vaccine von Elong Ngono, Annie, Syed, Thasneem, Nguyen, Anh-Viet, Regla-Nava, Jose Angel, Susantono, Mercylia, Spasova, Darina, Aguilar, Allison, West, Melissa, Sparks, Jessica, Gonzalez, Andrew, Branche, Emilie, DeHart, Jason L, Vega, Jerel Boyd, Karmali, Priya Prakash, Chivukula, Padmanabh, Kamrud, Kurt, Aliahmad, Parinaz, Wang, Nathaniel, Shresta, Sujan

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Effect of molecular weight of PGG–paclitaxel conjugates on in vitro and in vivo efficacy von Yang, Danbo, Liu, Xiaoqing, Jiang, Xinguo, Liu, Yun, Ying, Wenbin, Wang, Hai, Bai, Hao, Taylor, Wendy D., Wang, Yuwei, Clamme, Jean-Pierre, Co, Erick, Chivukula, Padmanabh, Tsang, Kwok Yin, Jin, Yi, Yu, Lei

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Hepatocyte-specific activity of TSC22D4 triggers progressive NAFLD by impairing mitochondrial function von Wolff, Gretchen, Sakurai, Minako, Mhamane, Amit, Troullinaki, Maria, Maida, Adriano, Deligiannis, Ioannis K., Yin, Kelvin, Weber, Peter, Morgenstern, Jakob, Wieder, Annika, Kwon, Yun, Sekar, Revathi, Zeigerer, Anja, Roden, Michael, Blüher, Matthias, Volk, Nadine, Poth, Tanja, Hackert, Thilo, Wiedmann, Lena, De Angelis Rigotti, Francesca, Rodriguez-Vita, Juan, Fischer, Andreas, Mukthavaram, Rajesh, Limphong, Pattraranee, Tachikawa, Kiyoshi, Karmali, Priya, Payne, Joseph, Chivukula, Padmanabh, Ekim-Üstünel, Bilgen, Martinez-Jimenez, Celia P., Szendrödi, Julia, Nawroth, Peter, Herzig, Stephan

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Abstract 4291: An optimized therapeutic nanoparticle delivery platform of miRNA in preclinical murine models of malignancy von Yaghi, Nasser K., Wei, Jun, Kong, Ling-Yuan, Hashimoto, Yuuri, Nduom, Edjah K., Huang, Neal, Ling, Xiaoyang, Zhou, Shouhao, Levine, Jonathan M., Fajt, Virginia R., Tachikawa, Kiyoshi, Chivukula, Padmanabh, Webb, David C., Payne, Joseph E., Heimberger, Amy B.

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BMET-14STAT3 INHIBITION ENHANCES THERAPEUTIC EFFICACY OF RADIATION TREATMENT AGAINST ESTABLISHED BRAIN METASTASIS IN MURINE MELANOMA MODEL von Hashimoto, Yuuri, Yaghi, Nasser K., Wei, Jun, Huang, Neal, Ezhilarasan, Ravesanker, Kong, Ling-Yuan, Zhou, Shouhao, Chivukula, Padmanabh, Webb, David C., Priebe, Waldemar, Payne, Joseph E., Sulman, Erik P., Heimberger, Amy B.

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IMPS-41IMMUNE MODULATORY NANOPARTICLE THERAPEUTICS von Yaghi, Nasser, Wei, Jun, Hashimoto, Yuuri, Kong, Ling-Yuan, Gabrusiewicz, Konrad, Nduom, Edjah, Ling, Xiaoyang, Huang, Neal, Zhou, Shouhao, Levine, Jonathan, Fajt, Virginia, Levine, Gwendolyn, Porter, Brian, Tachikawa, Kiyoshi, Chivukula, Padmanabh, Webb, David, Payne, Joseph, Heimberger, Amy

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IONIZABLE CATIONIC LIPID FOR RNA DELIVERY von CHIVUKULA, Padmanabh, PAYNE, Joseph

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