Funded Research Projects

2008

Characterization of post-transcriptional control mechanisms regulating SMN2 gene expression

Principal investigator(s):

Dr. Margarida Gama Carvalho

2008

Development of a stem cell approach for treating Spinal Muscular Atrophy

Principal investigator(s):

Dr. Giacomo Pietro Comi

2008

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

Principal investigator(s):

Dr. Martine Barkats

2008

Outcome measures in SMA types II and III

Principal investigator(s):

Professor Eugenio Mercuri