Funded Research Projects

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

2011

mRNA transport in SMA pathogenesis

Principal investigator(s):

Dr. Tilman Achsel

2010

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

Principal investigator(s):

Dr. Martine Barkats

2010

Delivery and correction of SMA using AAV vectors

Principal investigator(s):

Dr. Sandra Duqué

2010

Evalation of the contribution of SMN1 and SMN2 sequence variants to the clinical severity of SMA

Principal investigator(s):

Dr. Kathleen Mahias

2010

New drugs and new drug targets for Spinal Muscular Atrophy using chemical and functional genomics

Principal investigator(s):

Professor David Sattelle