A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy in a Mouse Model of SMARD1

por Shababi, Monir, Villalón, Eric, Kaifer, Kevin A., DeMarco, Vince, Lorson, Christian L.
Publicado 2018

Development of a Single Vector System that Enhances Trans-Splicing of SMN2 Transcripts

por Coady, Tristan H., Baughan, Travis D., Shababi, Monir, Passini, Marco A., Lorson, Christian L.
Publicado 2008

Defining the optimal dose and therapeutic window in SMA with respiratory distress type I model mice, FVB/NJ-Ighmpb2(nmd-2J)

por Shababi, Monir, Smith, Caley E., Ricardez Hernandez, Sara M., Marquez, Jose, Al Rawi, Zayd, Villalón, Eric, Farris, K. David, Garro-Kacher, Mona O., Lorson, Christian L.
Publicado 2021