Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy

Tekijä Zhao, Xin, Feng, Zhihua, Ling, Karen K. Y., Mollin, Anna, Sheedy, Josephine, Yeh, Shirley, Petruska, Janet, Narasimhan, Jana, Dakka, Amal, Welch, Ellen M., Karp, Gary, Chen, Karen S., Metzger, Friedrich, Ratni, Hasane, Lotti, Francesco, Tisdale, Sarah, Naryshkin, Nikolai A., Pellizzoni, Livio, Paushkin, Sergey, Ko, Chien-Ping, Weetall, Marla
Julkaisussa Hum Mol Genet (2016)

ELP1 Splicing Correction Reverses Proprioceptive Sensory Loss in Familial Dysautonomia

Tekijä Morini, Elisabetta, Gao, Dadi, Montgomery, Connor M., Salani, Monica, Mazzasette, Chiara, Krussig, Tobias A., Swain, Brooke, Dietrich, Paula, Narasimhan, Jana, Gabbeta, Vijayalakshmi, Dakka, Amal, Hedrick, Jean, Zhao, Xin, Weetall, Marla, Naryshkin, Nikolai A., Wojtkiewicz, Gregory G., Ko, Chien-Ping, Talkowski, Michael E., Dragatsis, Ioannis, Slaugenhaupt, Susan A.
Julkaisussa Am J Hum Genet (2019)

The DHODH Inhibitor PTC299 Arrests SARS-CoV-2 Replication and Suppresses Induction of Inflammatory Cytokines

Tekijä Luban, Jeremy, Sattler, Rachel, Mühlberger, Elke, Graci, Jason D., Cao, Liangxian, Weetall, Marla, Trotta, Christopher, Colacino, Joseph M., Bavari, Sina, Strambio-De-Castillia, Caterina, Suder, Ellen L., Wang, Yetao, Soloveva, Veronica, Cintron-Lue, Katherine, Naryshkin, Nikolai A., Pykett, Mark, Welch, Ellen M., O’Keefe, Kylie, Kong, Ronald, Goodwin, Elizabeth, Jacobson, Allan, Paessler, Slobodan, Peltz, Stuart
Julkaisussa bioRxiv (2020)

The DHODH inhibitor PTC299 arrests SARS-CoV-2 replication and suppresses induction of inflammatory cytokines

Tekijä Luban, Jeremy, Sattler, Rachel A., Mühlberger, Elke, Graci, Jason D., Cao, Liangxian, Weetall, Marla, Trotta, Christopher, Colacino, Joseph M., Bavari, Sina, Strambio-De-Castillia, Caterina, Suder, Ellen L., Wang, Yetao, Soloveva, Veronica, Cintron-Lue, Katherine, Naryshkin, Nikolai A., Pykett, Mark, Welch, Ellen M., O’Keefe, Kylie, Kong, Ronald, Goodwin, Elizabeth, Jacobson, Allan, Paessler, Slobodan, Peltz, Stuart W.
Julkaisussa Virus Res (2021)

Impaired prenatal motor axon development necessitates early therapeutic intervention in severe SMA

Tekijä Kong, Lingling, Valdivia, David O., Simon, Christian M., Hassinan, Cera W., Delestrée, Nicolas, Ramos, Daniel M., Park, Jae Hong, Pilato, Celeste M., Xu, Xixi, Crowder, Melissa, Grzyb, Chloe C., King, Zachary A., Petrillo, Marco, Swoboda, Kathryn J., Davis, Crystal, Lutz, Cathleen M., Stephan, Alexander H., Zhao, Xin, Weetall, Marla, Naryshkin, Nikolai A., Crawford, Thomas O., Mentis, George Z., Sumner, Charlotte J.
Julkaisussa Sci Transl Med (2021)