Concept

Atrophie

Publications associées (14)

Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6

Patrick Aebischer, Bernard Schneider, Christopher Towne

Retrograde transport of viral vectors in the rodent spinal cord provides a powerful means to administer a therapeutic transgene from the innervated musculature. With the aim of scaling up this approach to non-human primates, we have injected recombinant ad ...

2010

Adeno-Associated Virus-Mediated Gene Delivery for the Treatment of Amyotrophic Lateral Sclerosis and other Motor Neuron Disorders

Christopher Towne

The delivery of molecules and genes to the central nervous system (CNS) poses a major challenge for the treatment of neurodegenerative diseases. CNS disorders require long-term intervention and the presence of the blood-brain barrier (BBB) restricts the pe ...

EPFL2010

Rescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulation

Didier Trono, Isabelle Barde, Sonia Verp

In spinal muscular atrophy (SMA), the leading genetic cause of early childhood death, the survival motor neuron 1 gene (SMN1) is deleted or inactivated. The nearly identical SMN2 gene has a silent mutation that impairs the utilization of exon 7 and the pro ...

Oxford University Press2008

Complete and long-term rescue of lesioned adult motoneurons by lentiviral-mediated expression of glial cell line-derived neurotrophic factor in the facial nucleus

Patrick Aebischer

To date, delivery of neurotrophic factors has only allowed to transiently protect axotomized facial motoneurons against cell death. In the present report, long-term protection of these neurons was evaluated by continuously expressing the neurotrophic facto ...

2000

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