What Genetics Has Told Us and How It Can Inform Future Experiments for Spinal Muscular Atrophy, a Perspective

por Blatnik, Anton J., McGovern, Vicki L., Burghes, Arthur H. M.
Publicado 2021

Plastin 3 Expression Does Not Modify Spinal Muscular Atrophy Severity in the ∆7 SMA Mouse

por McGovern, Vicki L., Massoni-Laporte, Aurélie, Wang, Xueyong, Le, Thanh T., Le, Hao T., Beattie, Christine E., Rich, Mark M., Burghes, Arthur H. M.
Publicado 2015

In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans

por Schwartz, Maura K., Likhite, Shibi, Vetter, Tatyana A., Baird, Megan C., McGovern, Vicki, Sierra Delgado, Andrea, Mendel, Tom, Burghes, Arthur, Meyer, Kathrin C.
Publicado 2023

Electrophysiological biomarkers in spinal muscular atrophy: proof of concept

por David Arnold, W, Porensky, Paul N, McGovern, Vicki L, Iyer, Chitra C, Duque, Sandra, Li, Xiaobai, Meyer, Kathrin, Schmelzer, Leah, Kaspar, Brian K, Kolb, Stephen J, Kissel, John T, Burghes, Arthur H M
Publicado 2014

Normalization of Patient-Identified Plasma Biomarkers in SMNΔ7 Mice following Postnatal SMN Restoration

por Arnold, W. David, Duque, Sandra, Iyer, Chitra C., Zaworski, Phillip, McGovern, Vicki L., Taylor, Shannon J., von Herrmann, Katharine M., Kobayashi, Dione T., Chen, Karen S., Kolb, Stephen J., Paushkin, Sergey V., Burghes, Arthur H. M.
Publicado 2016

Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study

por Kolb, Stephen J., Coffey, Christopher S., Yankey, Jon W., Krosschell, Kristin, Arnold, W. David, Rutkove, Seward B., Swoboda, Kathryn J., Reyna, Sandra P., Sakonju, Ai, Darras, Basil T., Shell, Richard, Kuntz, Nancy, Castro, Diana, Iannaccone, Susan T., Parsons, Julie, Connolly, Anne M., Chiriboga, Claudia A., McDonald, Craig, Burnette, W. Bryan, Werner, Klaus, Thangarajh, Mathula, Shieh, Perry B., Finanger, Erika, Cudkowicz, Merit E., McGovern, Michelle M., McNeil, D. Elizabeth, Finkel, Richard, Kaye, Edward, Kingsley, Allison, Renusch, Samantha R., McGovern, Vicki L., Wang, Xueqian, Zaworski, Phillip G., Prior, Thomas W., Burghes, Arthur H.M., Bartlett, Amy, Kissel, John T.
Publicado 2016