Publications de Christopher Towne

SOD1 silencing in motoneurons or glia rescues neuromuscular function in ALS mice

Patrick Aebischer, Bernard Schneider, Cylia Cloé Rochat, Christopher Towne, Julianne Bobela, Elisabeth Anne Dirren

Objective: Amyotrophic lateral sclerosis is an incurable disorder mainly characterized by motoneuron degeneration. Mutations in the superoxide dismutase 1 (SOD1) gene account for 20% of familial forms of the disease. Mutant SOD1 exerts multiple pathogenic ...

Wiley-Blackwell2015

Intracerebroventricular Injection of Adeno-Associated Virus 6 and 9 Vectors for Cell Type-Specific Transgene Expression in the Spinal Cord

Patrick Aebischer, Bernard Schneider, Christopher Towne, Veronica Setola, Elisabeth Anne Dirren

In the context of motoneuron diseases, gene delivery as an experimental or therapeutic approach is hindered by the challenge to specifically target cell populations that are widely distributed along the spinal cord. Further complicating the task, transgene ...

Mary Ann Liebert2014

Neuroprotection by Gene Therapy Targeting Mutant SOD1 in Individual Pools of Motor Neurons Does not Translate Into Therapeutic Benefit in fALS Mice

Patrick Aebischer, Bernard Schneider, Christopher Towne, Veronica Setola

A major challenge in neurological gene therapy is delivery of the transgene to sufficient cell numbers in an atraumatic manner. This is particularly difficult for motor neuron (MN) diseases that have cells located across the entire spinal cord, brain stem, ...

Elsevier2010

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

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

Recombinant adeno-associated virus serotype 6 (rAAV2/6)-mediated gene transfer to nociceptive neurons through different routes of delivery

Patrick Aebischer, Christopher Towne

BACKGROUND: Gene transfer to nociceptive neurons of the dorsal root ganglia (DRG) is a promising approach to dissect mechanisms of pain in rodents and is a potential therapeutic strategy for the treatment of persistent pain disorders such as neuropathic pa ...

2009

Characterization of Nedd4-2 ubiquitin ligase expression in experimental neuropathic pain

Christopher Towne

2009

Regulation of Episomal Gene Expression by KRAB/KAP1-Mediated Histone Modifications

Patrick Aebischer, Vivianne Padrun, Christopher Towne, Didier Trono, Isabelle Barde, Sonia Verp, Anna Claire Groner

KAP1 is an essential cofactor of KRAB zinc finger proteins, a family of vertebrate-specific epigenetic repressors of largely unknown functions encoded in the hundreds by the mouse and human genomes. So far, KRAB/KAP1-mediated gene regulation has been studi ...

American Society for Microbiology2009

Lentiviral and Adeno-Associated Vector-Based Therapy for Motor Neuron Disease Through RNAi

Patrick Aebischer, Christopher Towne

RNAi holds promise for neurodegenerative disorders caused by gain-of-function mutations. We and others have demonstrated proof-of-principle for viral-mediated RNAi in a mouse model of motor neuron disease. Lentivirus and adeno-associated virus have been us ...

2009

Feasibility of gene therapy for the treatment of neuropathic pain: downregulation of GTP cyclohydrolase 1 expression via RNA interference

Patrick Aebischer, Christopher Towne

2008

Neuropathic pain: Gene therapy as a tool to transduce nociceptors

Patrick Aebischer, Christopher Towne

2008

Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice

Patrick Aebischer, Bernard Schneider, Christopher Towne

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder arising from the selective death of motor neurons. Approximately 20% of familial ALS (fALS) cases are caused by toxic gain-of-function mutations in the superoxide dismutase 1 (SOD1) ...

2008