Funded Research Projects

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

2011

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

Principal investigator(s):

Professor Matthew Wood

2011

Investigating novel genetic determinants of the SMA phenotype

Principal investigator(s):

Dr. Umrao Monani

2011

Spinal Muscular Atrophy: Molecular and functional analysis of a new modifier of SMA

Principal investigator(s):

Dr. Markus Riessland