siRNA carrying an (E)-vinylphosphonate moiety at the 5΄ end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2

por Elkayam, Elad, Parmar, Rubina, Brown, Christopher R., Willoughby, Jennifer L., Theile, Christopher S., Manoharan, Muthiah, Joshua-Tor, Leemor
Publicado 2017

siRNA carrying an (E)-vinylphosphonate moiety at the 5΄ end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2

por Elkayam, Elad, Parmar, Rubina, Brown, Christopher R., Willoughby, Jennifer L., Theile, Christopher S., Manoharan, Muthiah, Joshua-Tor, Leemor
Publicado 2017

Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy

por Engelbeen, Sarah, Pasteuning-Vuhman, Svetlana, Boertje-van der Meulen, Joke, Parmar, Rubina, Charisse, Klaus, Sepp-Lorenzino, Laura, Manoharan, Muthiah, Aartsma-Rus, Annemieke, van Putten, Maaike
Publicado 2023

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

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