Funded Research Projects

2010

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

Principal investigator(s):

Dr. Steve Wilton

2010

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

Principal investigator(s):

Dr. Béatrice Joussemet

2010

Gene therapy for Spinal Muscular Atrophy by a correction of SMN2 MRNA splicing

Principal investigator(s):

Dr. Rachel Nlend Nlend

2010

Invastigating the temporal requirements of the SMN protein in Spinal Muscular Atrophy

Principal investigator(s):

Dr. Umrao Monani

2010

PTEN modulation effects on motor neuron axonal growth and neuromuscular junction size in Spinal Muscular Atrophy

Principal investigator(s):

Professor Mimoun Azzouz

2010

The S. pombe model organism: tool to find suppressors of snRNP-mediated splicing defects”

Principal investigator(s):

Dr. Remy Bordonné

2009

A two-pronged approach to develop a treatment for Spinal Muscular Atrophy

Principal investigator(s):

Dr. Monir Shababi

2009

Characterization of post-transcriptional control mechanisms regulating SMN2 gene expression

Principal investigator(s):

Dr. Margarida Gama Carvalho