Funded Research Projects

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

2012

Evaluation of the contribution of SMN1 and SMN2 sequence variants to the clinical severity of SMADr. Kathleen Mahias

Principal investigator(s):

Dr. Kathleen Mahias

2012

Investigating novel genetic determinants of the SMA phenotype

Principal investigator(s):

Dr. Umrao Monani

2012

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

Principal investigator(s):

Dr. Markus Riessland

2012

mRNA transport in SMA pathogenesis

Principal investigator(s):

Dr. Tilman Achsel

2011

AAV9-mediated gene therapy in murine and feline models of SMA

Principal investigator(s):

Dr. Martine Barkats