Funded Research Projects

2014

Study of the role of SMN in the regulation of muscle-resident progenitors for the identification of novel therapeutic targets for SMA

Principal investigator(s):

Dr. Martine Barkats

2013

Cellular basis of motor circuit dysfunction induced by SMN deficiency

Principal investigator(s):

Dr. George Mentis

2013

Modulating SMN2 splicing with dual inhibitors of Sam68 and hnRNP A1: A novel therapeutic approach for Spinal Muscular Atrophy

Principal investigator(s):

Professor Eduardo Tizzano

2013

Using RNA secondary structure as a therapeutic target for Spinal Muscular Atrophy: a new drug discovery approach

Principal investigator(s):

Dr. Amparo Garcia-Lopez

2012

Elucidate the molecular mechanism underlying maturation and remodelling defects of the neuromuscular system in SMA

Principal investigator(s):

Dr. Shingo Kariya

2012

In search of small molecules targeting protein-RNA complex: a novel approach against Spinal Muscular Atrophy

Principal investigator(s):

Dr. Frédéric Allain

2012

Optimization of AAV9-SMN gene therapy for SMA

Principal investigator(s):

Dr. Martine Barkats

2011

AAV9- mediated gene therapy in murine models of SMA

Principal investigator(s):

Dr. Yannick Tanguy