Funded Research Projects

2009

Characterization of post-transcriptional control mechanisms regulating SMN2 gene expression

Principal investigator(s):

Dr. Margarida Gama Carvalho

2009

Development of a stem cell approach for treating Spinal Muscular Atrophy

Principal investigator(s):

Dr. Giacomo Pietro Comi

2009

Evaluation of AAV-mediated gene therapy in murine and feline SMA models

Principal investigator(s):

Dr. Martine Barkats

2009

Outcome measures in SMA types II and III

Principal investigator(s):

Professor Eugenio Mercuri