The Nonclinical Safety Profile of GalNAc-conjugated RNAi Therapeutics in Subacute Studies

por Janas, Maja M., Harbison, Carole E., Perry, Victoria K., Carito, Brenda, Sutherland, Jessica E., Vaishnaw, Akshay K., Keirstead, Natalie D., Warner, Garvin
Publicado 2018

Impact of Serum Proteins on the Uptake and RNAi Activity of GalNAc-Conjugated siRNAs

por Agarwal, Saket, Allard, Ruth, Darcy, Justin, Chigas, Samantha, Gu, Yongli, Nguyen, Tuyen, Bond, Sarah, Chong, Saeho, Wu, Jing-Tao, Janas, Maja M.
Publicado 2021

Feed-Forward Microprocessing and Splicing Activities at a MicroRNA–Containing Intron

por Janas, Maja M., Khaled, Mehdi, Schubert, Steffen, Bernstein, Jacob G., Golan, David, Veguilla, Rosa A., Fisher, David E., Shomron, Noam, Levy, Carmit, Novina, Carl D.
Publicado 2011

Chimeric siRNAs with chemically modified pentofuranose and hexopyranose nucleotides: altritol-nucleotide (ANA) containing GalNAc–siRNA conjugates: in vitro and in vivo RNAi activit...

por Kumar, Pawan, Degaonkar, Rohan, Guenther, Dale C, Abramov, Mikhail, Schepers, Guy, Capobianco, Marie, Jiang, Yongfeng, Harp, Joel, Kaittanis, Charalambos, Janas, Maja M, Castoreno, Adam, Zlatev, Ivan, Schlegel, Mark K, Herdewijn, Piet, Egli, Martin, Manoharan, Muthiah
Publicado 2020

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

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

siRNAs containing 2′-fluorinated Northern-methanocarbacyclic (2′-F-NMC) nucleotides: in vitro and in vivo RNAi activity and inability of mitochondrial polymerases to incorporate 2′...

por Akabane-Nakata, Masaaki, Erande, Namrata D, Kumar, Pawan, Degaonkar, Rohan, Gilbert, Jason A, Qin, June, Mendez, Martha, Woods, Lauren Blair, Jiang, Yongfeng, Janas, Maja M, O’Flaherty, Derek K, Zlatev, Ivan, Schlegel, Mark K, Matsuda, Shigeo, Egli, Martin, Manoharan, Muthiah
Publicado 2021

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

Safety evaluation of 2′-deoxy-2′-fluoro nucleotides in GalNAc-siRNA conjugates

por Janas, Maja M, Zlatev, Ivan, Liu, Ju, Jiang, Yongfeng, Barros, Scott A, Sutherland, Jessica E, Davis, Wendell P, Liu, Jingxuan, Brown, Christopher R, Liu, Xiumin, Schlegel, Mark K, Blair, Lauren, Zhang, Xuemei, Das, Biplab, Tran, Chris, Aluri, Krishna, Li, Jing, Agarwal, Saket, Indrakanti, Ramesh, Charisse, Klaus, Nair, Jayaprakash, Matsuda, Shigeo, Rajeev, Kallanthottathil G, Zimmermann, Tracy, Sepp-Lorenzino, Laura, Xu, Yuanxin, Akinc, Akin, Fitzgerald, Kevin, Vaishnaw, Akshay K, Smith, Peter F, Manoharan, Muthiah, Jadhav, Vasant, Wu, Jing-Tao, Maier, Martin A
Publicado 2019