An investigational RNAi therapeutic targeting Factor XII (ALN-F12) for the treatment of hereditary angioedema

por Liu, Jingxuan, Qin, June, Borodovsky, Anna, Racie, Timothy, Castoreno, Adam, Schlegel, Mark, Maier, Martin A., Zimmerman, Tracy, Fitzgerald, Kevin, Butler, James, Akinc, Akin
Publicado 2019

Generation and validation of structurally defined antibody–siRNA conjugates

por Nanna, Alex R, Kel’in, Alexander V, Theile, Christopher, Pierson, Justin M, Voo, Zhi Xiang, Garg, Ashish, Nair, Jayaprakash K, Maier, Martin A, Fitzgerald, Kevin, Rader, Christoph
Publicado 2020

Crystal structure, stability and in vitro RNAi activity of oligoribonucleotides containing the ribo-difluorotoluyl nucleotide: insights into substrate requirements by the human RIS...

por Li, Feng, Pallan, Pradeep S., Maier, Martin A., Rajeev, Kallanthottathil G., Mathieu, Steven L., Kreutz, Christoph, Fan, Yupeng, Sanghvi, Jayodita, Micura, Ronald, Rozners, Eriks, Manoharan, Muthiah, Egli, Martin
Publicado 2007

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

por Foster, Donald J., Brown, Christopher R., Shaikh, Sarfraz, Trapp, Casey, Schlegel, Mark K., Qian, Kun, Sehgal, Alfica, Rajeev, Kallanthottathil G., Jadhav, Vasant, Manoharan, Muthiah, Kuchimanchi, Satya, Maier, Martin A., Milstein, Stuart
Publicado 2018

Overcoming GNA/RNA base-pairing limitations using isonucleotides improves the pharmacodynamic activity of ESC+ GalNAc-siRNAs

por Schlegel, Mark K, Matsuda, Shigeo, Brown, Christopher R, Harp, Joel M, Barry, Joseph D, Berman, Daniel, Castoreno, Adam, Schofield, Sally, Szeto, John, Manoharan, Muthiah, Charissé, Klaus, Egli, Martin, Maier, Martin A
Publicado 2021

Structural basis for the synergy of 4′- and 2′-modifications on siRNA nuclease resistance, thermal stability and RNAi activity

por Harp, Joel M, Guenther, Dale C, Bisbe, Anna, Perkins, Lydia, Matsuda, Shigeo, Bommineni, Gopal R, Zlatev, Ivan, Foster, Donald J, Taneja, Nate, Charisse, Klaus, Maier, Martin A, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Egli, Martin
Publicado 2018

Modulation of thermal stability can enhance the potency of siRNA

por Addepalli, Haripriya, Meena, Peng, Chang G., Wang, Gang, Fan, Yupeng, Charisse, Klaus, Jayaprakash, K. Narayanannair, Rajeev, Kallanthottathil G., Pandey, Rajendra K., Lavine, Gary, Zhang, Ligang, Jahn-Hofmann, Kerstin, Hadwiger, Philipp, Manoharan, Muthiah, Maier, Martin A.
Publicado 2010

Role of a “Magic” Methyl: 2′-Deoxy-2′-α-F-2′-β-C-methyl Pyrimidine Nucleotides Modulate RNA Interference Activity through Synergy with 5′-Phosphate Mimics and Mitigation of Off-Tar...

por Guenther, Dale C., Mori, Shohei, Matsuda, Shigeo, Gilbert, Jason A., Willoughby, Jennifer L. S., Hyde, Sarah, Bisbe, Anna, Jiang, Yongfeng, Agarwal, Saket, Madaoui, Mimouna, Janas, Maja M., Charisse, Klaus, Maier, Martin A., Egli, Martin, Manoharan, Muthiah
Publicado 2022

Identification of siRNA delivery enhancers by a chemical library screen

por Gilleron, Jerome, Paramasivam, Prasath, Zeigerer, Anja, Querbes, William, Marsico, Giovanni, Andree, Cordula, Seifert, Sarah, Amaya, Pablo, Stöter, Martin, Koteliansky, Victor, Waldmann, Herbert, Fitzgerald, Kevin, Kalaidzidis, Yannis, Akinc, Akin, Maier, Martin A., Manoharan, Muthiah, Bickle, Marc, Zerial, Marino
Publicado 2015

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

por Janas, Maja M., Schlegel, Mark K., Harbison, Carole E., Yilmaz, Vedat O., Jiang, Yongfeng, Parmar, Rubina, Zlatev, Ivan, Castoreno, Adam, Xu, Huilei, Shulga-Morskaya, Svetlana, Rajeev, Kallanthottathil G., Manoharan, Muthiah, Keirstead, Natalie D., Maier, Martin A., Jadhav, Vasant
Publicado 2018

Centyrin ligands for extrahepatic delivery of siRNA

por Klein, Donna, Goldberg, Shalom, Theile, Christopher S., Dambra, Richard, Haskell, Kathleen, Kuhar, Elise, Lin, Tricia, Parmar, Rubina, Manoharan, Muthiah, Richter, Mark, Wu, Meizhen, Mendrola Zarazowski, Jeannine, Jadhav, Vasant, Maier, Martin A., Sepp-Lorenzino, Laura, O’Neil, Karyn, Dudkin, Vadim
Publicado 2021

Chirality matters: stereo-defined phosphorothioate linkages at the termini of small interfering RNAs improve pharmacology in vivo

por Jahns, Hartmut, Taneja, Nate, Willoughby, Jennifer L S, Akabane-Nakata, Masaaki, Brown, Christopher R, Nguyen, Tuyen, Bisbe, Anna, Matsuda, Shigeo, Hettinger, Matt, Manoharan, Rajar M, Rajeev, Kallanthottathil G, Maier, Martin A, Zlatev, Ivan, Charisse, Klaus, Egli, Martin, Manoharan, Muthiah
Publicado 2021

Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles

por Mui, Barbara L, Tam, Ying K, Jayaraman, Muthusamy, Ansell, Steven M, Du, Xinyao, Tam, Yuen Yi C, Lin, Paulo JC, Chen, Sam, Narayanannair, Jayaprakash K, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Akinc, Akin, Maier, Martin A, Cullis, Pieter, Madden, Thomas D, Hope, Michael J
Publicado 2013

Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression

por Willoughby, Jennifer L.S., Chan, Amy, Sehgal, Alfica, Butler, James S., Nair, Jayaprakash K., Racie, Tim, Shulga-Morskaya, Svetlana, Nguyen, Tuyen, Qian, Kun, Yucius, Kristina, Charisse, Klaus, van Berkel, Theo J.C., Manoharan, Muthiah, Rajeev, Kallanthottathil G., Maier, Martin A., Jadhav, Vasant, Zimmermann, Tracy S.
Publicado 2018

Small circular interfering RNAs (sciRNAs) as a potent therapeutic platform for gene-silencing

por Jahns, Hartmut, Degaonkar, Rohan, Podbevsek, Peter, Gupta, Swati, Bisbe, Anna, Aluri, Krishna, Szeto, John, Kumar, Pawan, LeBlanc, Sarah, Racie, Tim, Brown, Christopher R, Castoreno, Adam, Guenther, Dale C, Jadhav, Vasant, Maier, Martin A, Plavec, Janez, Egli, Martin, Manoharan, Muthiah, Zlatev, Ivan
Publicado 2021

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

por Jayaraman, Muthusamy, Ansell, Steven M, Mui, Barbara L, Tam, Ying K, Chen, Jianxin, Du, Xinyao, Butler, David, Eltepu, Laxman, Matsuda, Shigeo, Narayanannair, Jayaprakash K, Rajeev, Kallanthottathil G, Hafez, Ismail M, Akinc, Akin, Maier, Martin A, Tracy, Mark A, Cullis, Pieter R, Madden, Thomas D, Manoharan, Muthiah, Hope, Michael J
Publicado 2012

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

por Nair, Jayaprakash K., Attarwala, Husain, Sehgal, Alfica, Wang, Qianfan, Aluri, Krishna, Zhang, Xuemei, Gao, Minggeng, Liu, Ju, Indrakanti, Ramesh, Schofield, Sally, Kretschmer, Philip, Brown, Christopher R., Gupta, Swati, Willoughby, Jennifer L.S., Boshar, Julie A., Jadhav, Vasant, Charisse, Klaus, Zimmermann, Tracy, Fitzgerald, Kevin, Manoharan, Muthiah, Rajeev, Kallanthottathil G., Akinc, Akin, Hutabarat, Renta, Maier, Martin A.
Publicado 2017

Investigating the pharmacodynamic durability of GalNAc–siRNA conjugates

por Brown, Christopher R, Gupta, Swati, Qin, June, Racie, Timothy, He, Guo, Lentini, Scott, Malone, Ryan, Yu, Mikyung, Matsuda, Shigeo, Shulga-Morskaya, Svetlana, Nair, Anil V, Theile, Christopher S, Schmidt, Karyn, Shahraz, Azar, Goel, Varun, Parmar, Rubina G, Zlatev, Ivan, Schlegel, Mark K, Nair, Jayaprakash K, Jayaraman, Muthusamy, Manoharan, Muthiah, Brown, Dennis, Maier, Martin A, Jadhav, Vasant
Publicado 2020

RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes

por Subramanian, Megha, McIninch, James, Zlatev, Ivan, Schlegel, Mark K., Kaittanis, Charalambos, Nguyen, Tuyen, Agarwal, Saket, Racie, Timothy, Alvarado, Martha Arbaiza, Wassarman, Kelly, Collins, Thomas S., Chickering, Tyler, Brown, Christopher R., Schmidt, Karyn, Castoreno, Adam B., Shulga-Morskaya, Svetlana, Stamenova, Elena, Buckowing, Kira, Berman, Daniel, Barry, Joseph D., Bisbe, Anna, Maier, Martin A., Fitzgerald, Kevin, Jadhav, Vasant
Publicado 2023

From bench to bedside: Improving the clinical safety of GalNAc–siRNA conjugates using seed-pairing destabilization

por Schlegel, Mark K, Janas, Maja M, Jiang, Yongfeng, Barry, Joseph D, Davis, Wendell, Agarwal, Saket, Berman, Daniel, Brown, Christopher R, Castoreno, Adam, LeBlanc, Sarah, Liebow, Abigail, Mayo, Tara, Milstein, Stuart, Nguyen, Tuyen, Shulga-Morskaya, Svetlana, Hyde, Sarah, Schofield, Sally, Szeto, John, Woods, Lauren Blair, Yilmaz, Vedat O, Manoharan, Muthiah, Egli, Martin, Charissé, Klaus, Sepp-Lorenzino, Laura, Haslett, Patrick, Fitzgerald, Kevin, Jadhav, Vasant, Maier, Martin A
Publicado 2022