Gene therapy for amyotrophic lateral sclerosis

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.

► Therapeutic target: motor neurons, glial, and/or skeletal muscles.
► Transgenic models of ALS.
► Gene delivery approaches to motor neurons.
► Therapeutic transgenes.
► Parallel approaches.