Acyclic (S)-glycol nucleic acid (S-GNA) modification of siRNAs improves the safety of RNAi therapeutics while maintaining potency

por Egli, Martin, Schlegel, Mark K., Manoharan, Muthiah
Publicado 2023

Duplex Structure of a Minimal Nucleic Acid

por Schlegel, Mark K., Essen, Lars-Oliver, Meggers, Eric
Publicado 2008

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

Synthesis, chirality-dependent conformational and biological properties of siRNAs containing 5′-(R)- and 5′-(S)-C-methyl-guanosine

por Mikami, Atsushi, Erande, Namrata, Matsuda, Shigeo, Kel’in, Alexander, Woods, Lauren Blair, Chickering, Tyler, Pallan, Pradeep S, Schlegel, Mark K, Zlatev, Ivan, Egli, Martin, Manoharan, Muthiah
Publicado 2020

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

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

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

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

In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene

por De Giorgi, Marco, Park, So Hyun, Castoreno, Adam, Cao, Mingming, Hurley, Ayrea, Saxena, Lavanya, Chuecos, Marcel A., Walkey, Christopher J., Doerfler, Alexandria M., Furgurson, Mia N., Ljungberg, M. Cecilia, Patel, Kalyani R., Hyde, Sarah, Chickering, Tyler, Lefebvre, Stephanie, Wassarman, Kelly, Miller, Patrick, Qin, June, Schlegel, Mark K., Zlatev, Ivan, Li, Rich Gang, Kim, Jong, Martin, James F., Bissig, Karl-Dimiter, Jadhav, Vasant, Bao, Gang, Lagor, William R.
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

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