title: 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.
authors: Emory University, Department of Neurosurgery, 1365B Clifton Road NE, Suite B6200, Atlanta, GA 30322, USA.
authors: Thais
authors: T
authors: Federici
authors: Nicholas M
authors: NM
authors: Boulis
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000453:epidemiology / Q000473:pathology / Q000628:therapy*; D000818:Animals; D030801:Animals, Genetically Modified; D001253:Astrocytes / Q000502:physiology; D064987:Cell- and Tissue-Based Therapy; D004195:Disease Models, Animal; D020868:Gene Silencing; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D009046:Motor Neurons / Q000502:physiology; D009134:Muscular Atrophy, Spinal / Q000628:therapy; D009469:Neuromuscular Junction / Q000502:physiology