Funded Research Projects

2014

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

2014

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

Principal investigator(s):

Dr. Amparo Garcia-Lopez

2013

AAV9-SMN Gene Therapy for SMA

Principal investigator(s):

Dr. Martine Barkats

2013

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

Principal investigator(s):

Dr. Shingo Kariya

2013

In search of small molecules targeting protein-RNA complex

Principal investigator(s):

Dr. Frédéric Allain

2013

Optimization of AAV9-SMN gene therapy for SMA

Principal investigator(s):

Dr. Martine Barkats

2012

AAV9- mediated gene therapy in murine models of SMA

Principal investigator(s):

Dr. Yannick Tanguy

2012

Delivery of splice switching oligonucleotides using exosomes for the treatment of spinal muscular atrophy

Principal investigator(s):

Professor Matthew Wood