Structure-Based Designed Nano-Dysferlin Significantly Improves Dysferlinopathy in BLA/J Mice

Por Llanga, Telmo, Nagy, Nadia, Conatser, Laura, Dial, Catherine, Sutton, R. Bryan, Hirsch, Matthew L.
Publicado no Mol Ther (2017)

AAV Gene Therapy for MPS1-associated Corneal Blindness

Por Vance, Melisa, Llanga, Telmo, Bennett, Will, Woodard, Kenton, Murlidharan, Giridhar, Chungfat, Neil, Asokan, Aravind, Gilger, Brian, Kurtzberg, Joanne, Samulski, R. Jude, Hirsch, Matthew L.
Publicado no Sci Rep (2016)

Hip region muscular dystrophy and emergence of motor deficits in dysferlin‐deficient Bla/J mice

Por Nagy, Nadia, Nonneman, Randal J., Llanga, Telmo, Dial, Catherine F., Riddick, Natallia V., Hampton, Tom, Moy, Sheryl S., Lehtimäki, Kimmo K., Ahtoniemi, Toni, Puoliväli, Jukka, Windish, Hillarie, Albrecht, Douglas, Richard, Isabelle, Hirsch, Matthew L.
Publicado no Physiol Rep (2017)