RNAi Therapies: Drugging the Undruggable

Qili Chu, Christina L. Frey, Vladimir S. Trubetskoy, Julia Hegge, Darren Wakefield, David B. Rozema, David L. Lewis, So C. Wong, Jason J. Klein, Holly L. Hamilton, Co-Injection of a Targeted, Reversibly Masked Endosomolytic Polymer Dramatically Improves the Efficacy of Cholesterol-Conjugated Small Interfering RNAs In Vivo Nucleic Acid Therapeutics. ,vol. 22, pp. 380- 390 ,(2012) , 10.1089/NAT.2012.0389

Michael Steckel, Miriam Molina-Arcas, Britta Weigelt, Michaela Marani, Patricia H Warne, Hanna Kuznetsov, Gavin Kelly, Becky Saunders, Michael Howell, Julian Downward, David C Hancock, Determination of synthetic lethal interactions in KRAS oncogene-dependent cancer cells reveals novel therapeutic targeting strategies Cell Research. ,vol. 22, pp. 1227- 1245 ,(2012) , 10.1038/CR.2012.82

Sherry Y. Wu, Xianbin Yang, Kshipra M. Gharpure, Hiroto Hatakeyama, Martin Egli, Michael H. McGuire, Archana S. Nagaraja, Takahito M. Miyake, Rajesha Rupaimoole, Chad V. Pecot, Morgan Taylor, Sunila Pradeep, Malgorzata Sierant, Cristian Rodriguez-Aguayo, Hyun J. Choi, Rebecca A. Previs, Guillermo N. Armaiz-Pena, Li Huang, Carlos Martinez, Tom Hassell, Cristina Ivan, Vasudha Sehgal, Richa Singhania, Hee-Dong Han, Chang Su, Ji Hoon Kim, Heather J. Dalton, Chandra Kovvali, Khandan Keyomarsi, Nigel A. J. McMillan, Willem W. Overwijk, Jinsong Liu, Ju-Seog Lee, Keith A. Baggerly, Gabriel Lopez-Berestein, Prahlad T. Ram, Barbara Nawrot, Anil K. Sood, 2′-OMe-phosphorodithioate-modified siRNAs show increased loading into the RISC complex and enhanced anti-tumour activity Nature Communications. ,vol. 5, pp. 3459- 3459 ,(2014) , 10.1038/NCOMMS4459

Brice E. Keyes, Naoki Oshimori, Elaine Fuchs, Slobodan Beronja, Peter Janki, Evan Heller, Wen-Hui Lien, RNAi screens in mice identify physiological regulators of oncogenic growth. Nature. ,vol. 501, pp. 185- 190 ,(2013) , 10.1038/NATURE12464

Cheng-Qiong Mao, Meng-Hua Xiong, Yang Liu, Song Shen, Xiao-Jiao Du, Xian-Zhu Yang, Shuang Dou, Pei-Zhuo Zhang, Jun Wang, None, Synthetic Lethal Therapy for KRAS Mutant Non-small-cell Lung Carcinoma with Nanoparticle-mediated CDK4 siRNA Delivery Molecular Therapy. ,vol. 22, pp. 964- 973 ,(2014) , 10.1038/MT.2014.18

Mark E. Davis, Jonathan E. Zuckerman, Chung Hang J. Choi, David Seligson, Anthony Tolcher, Christopher A. Alabi, Yun Yen, Jeremy D. Heidel, Antoni Ribas, Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles Nature. ,vol. 464, pp. 1067- 1070 ,(2010) , 10.1038/NATURE08956

D. Schramek, A. Sendoel, J. P. Segal, S. Beronja, E. Heller, D. Oristian, B. Reva, E. Fuchs, Direct in Vivo RNAi Screen Unveils Myosin IIa as a Tumor Suppressor of Squamous Cell Carcinomas Science. ,vol. 343, pp. 309- 313 ,(2014) , 10.1126/SCIENCE.1248627

Christine I Wooddell, David B Rozema, Markus Hossbach, Matthias John, Holly L Hamilton, Qili Chu, Julia O Hegge, Jason J Klein, Darren H Wakefield, Claudia E Oropeza, Jochen Deckert, Ingo Roehl, Kerstin Jahn-Hofmann, Philipp Hadwiger, Hans-Peter Vornlocher, Alan McLachlan, David L Lewis, Hepatocyte-targeted RNAi Therapeutics for the Treatment of Chronic Hepatitis B Virus Infection Molecular Therapy. ,vol. 21, pp. 973- 985 ,(2013) , 10.1038/MT.2013.31

Dirk Strumberg, Beate Schultheis, Ulrich Traugott, Christiane Vank, Ansgar Santel, Oliver Keil, Klaus Giese, Jörg Kaufmann, Joachim Drevs, Phase I clinical development of Atu027, a siRNA formulation targeting PKN3 in patients with advanced solid tumors. principles and practice of constraint programming. ,vol. 50, pp. 76- 78 ,(2012) , 10.5414/CPP50076

Martin A Maier, Muthusamy Jayaraman, Shigeo Matsuda, Ju Liu, Scott Barros, William Querbes, Ying K Tam, Steven M Ansell, Varun Kumar, June Qin, Xuemei Zhang, Qianfan Wang, Sue Panesar, Renta Hutabarat, Mary Carioto, Julia Hettinger, Pachamuthu Kandasamy, David Butler, Kallanthottathil G Rajeev, Bo Pang, Klaus Charisse, Kevin Fitzgerald, Barbara L Mui, Xinyao Du, Pieter Cullis, Thomas D Madden, Michael J Hope, Muthiah Manoharan, Akin Akinc, Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics Molecular Therapy. ,vol. 21, pp. 1570- 1578 ,(2013) , 10.1038/MT.2013.124