Cell-Selective Adeno-Associated Virus-Mediated SCN1A Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model and Is Well Tolerated in Nonh...

por Tanenhaus, Annie, Stowe, Timothy, Young, Andrew, McLaughlin, John, Aeran, Rangoli, Lin, I. Winnie, Li, Jianmin, Hosur, Raghavendra, Chen, Ming, Leedy, Jennifer, Chou, Tiffany, Pillay, Sirika, Vila, Maria Candida, Kearney, Jennifer A., Moorhead, Martin, Belle, Archana, Tagliatela, Stephanie
Publicado 2022

Elusive sources of variability of dystrophin rescue by exon skipping

por Vila, Maria Candida, Klimek, Margaret Benny, Novak, James S., Rayavarapu, Sree, Uaesoontrachoon, Kitipong, Boehler, Jessica F., Fiorillo, Alyson A., Hogarth, Marshall W., Zhang, Aiping, Shaughnessy, Conner, Gordish-Dressman, Heather, Burki, Umar, Straub, Volker, Lu, Qi Long, Partridge, Terence A., Brown, Kristy J., Hathout, Yetrib, van den Anker, John, Hoffman, Eric P., Nagaraju, Kanneboyina
Publicado 2015

INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy

por Dowling, James J., Müller-Felber, Wolfgang, Smith, Barbara K., Bönnemann, Carsten G., Kuntz, Nancy L., Muntoni, Francesco, Servais, Laurent, Alfano, Lindsay N., Beggs, Alan H., Bilder, Deborah A., Blaschek, Astrid, Duong, Tina, Graham, Robert J., Jain, Minal, Lawlor, Michael W., Lee, Jun, Coats, Julie, Lilien, Charlotte, Lowes, Linda P., MacBean, Victoria, Neuhaus, Sarah, Noursalehi, Mojtaba, Pitts, Teresa, Finlay, Caroline, Christensen, Sarah, Rafferty, Gerrard, Seferian, Andreea M., Tsuchiya, Etsuko, James, Emma S., Miller, Weston, Sepulveda, Bryan, Vila, Maria Candida, Prasad, Suyash, Rico, Salvador, Shieh, Perry B.
Publicado 2022