Synaptic Defects in the Spinal and Neuromuscular Circuitry in a Mouse Model of Spinal Muscular Atrophy

por Ling, Karen K. Y., Lin, Ming-Yi, Zingg, Brian, Feng, Zhihua, Ko, Chien-Ping
Publicado 2010

Long-Term SMN- and Ncald-ASO Combinatorial Therapy in SMA Mice and NCALD-ASO Treatment in hiPSC-Derived Motor Neurons Show Protective Effects

por Muiños-Bühl, Anixa, Rombo, Roman, Ling, Karen K., Zilio, Eleonora, Rigo, Frank, Bennett, C. Frank, Wirth, Brunhilde
Publicado 2023

Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1

por Ait Benichou, Siham, Jauvin, Dominic, De Serres-Bérard, Thiéry, Pierre, Marion, Ling, Karen K., Bennett, C. Frank, Rigo, Frank, Gourdon, Genevieve, Chahine, Mohamed, Puymirat, Jack
Publicado 2022

Pathological impact of SMN2 mis-splicing in adult SMA mice

por Sahashi, Kentaro, Ling, Karen K Y, Hua, Yimin, Wilkinson, John Erby, Nomakuchi, Tomoki, Rigo, Frank, Hung, Gene, Xu, David, Jiang, Ya-Ping, Lin, Richard Z, Ko, Chien-Ping, Bennett, C Frank, Krainer, Adrian R
Publicado 2013

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

por 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
Publicado 2016

Mutant Profilin1 transgenic mice recapitulate cardinal features of motor neuron disease

por Fil, Daniel, DeLoach, Abigail, Yadav, Shilpi, Alkam, Duah, MacNicol, Melanie, Singh, Awantika, Compadre, Cesar M., Goellner, Joseph J., O’Brien, Charles A., Fahmi, Tariq, Basnakian, Alexei G., Calingasan, Noel Y., Klessner, Jodi L., Beal, Flint M., Peters, Owen M., Metterville, Jake, Brown, Robert H., Ling, Karen K.Y., Rigo, Frank, Ozdinler, P. Hande, Kiaei, Mahmoud
Publicado 2017