Funded Research Projects

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

2009

Analysis of mRNA transport and local protein syntheses in axons of SMA motor neurons

Principal investigator(s):

Dr. Claudia Fallini

2009

Antisense oligomer induced restoration of expression as a therapy for Spinal Muscular Atrophy

Principal investigator(s):

Dr. Steve Wilton

2009

Evaluation of new AAV-mediated gene therapy strategies in a feline model of Spinal Muscular Atrophy

Principal investigator(s):

Dr. Béatrice Joussemet