Funded Research Projects

2009

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

Principal investigator(s):

Dr. Rachel Nlend Nlend

2009

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

Principal investigator(s):

Dr. Umrao Monani

2009

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

Principal investigator(s):

Professor Mimoun Azzouz

2009

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

Principal investigator(s):

Dr. Remy Bordonné

2008

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

Principal investigator(s):

Dr. Monir Shababi

2008

Characterization of post-transcriptional control mechanisms regulating SMN2 gene expression

Principal investigator(s):

Dr. Margarida Gama Carvalho

2008

Development of a stem cell approach for treating Spinal Muscular Atrophy

Principal investigator(s):

Dr. Giacomo Pietro Comi

2008

Evaluation of AAV-mediated gene therapy in murine and feline SMA models

Principal investigator(s):

Dr. Martine Barkats