Gene therapy for amyotrophic lateral sclerosis

Abstract

Gene therapy continues to be a potential option for amyotrophic lateral sclerosis (ALS). This chapter will inform the reader about promising therapeutic transgenes and proof-of-principle studies in transgenic rodent models of ALS. Challenges regarding the disease targets and time for therapeutic intervention will be also discussed. Finally, restorative therapy for ALS, as well as gene therapy for other motor neuron diseases will be briefly reviewed.

Keywords

• Astrocytes
• Gene delivery
• Motor neurons
• Neurotrophic factors
• RNA interference
• Superoxide dismutase 1