ABSTRACT
Motor neuron diseases (MND) are clinically distinct neurodegenerative diseases sharing a primary disorder involving the motor system. MND encompass motor syndromes of amyotrophic lateral sclerosis (ALS), spinal muscular atrophy, X-linked spinobulbar muscular atrophy (Kennedy’s disease), hereditary spastic paraplegia, and postpolio muscular atrophy. MND are often lethal and incurable diseases that arise from relentless dysfunction of upper (motor cortical), lower (brainstem and spinal) or both motor neurons. Extensive research over the last decade has provided new insights into the genetic of a variety of motor neuron syndromes. A better understanding of the molecular events leading to motor neuron death has stimulated new therapeutic concepts including gene therapy. The development of transgenic animal models, which clinically recapitulate human MND, also provided more relevant targets to test novel therapeutic approaches. Due to the molecular complexity of these diseases and the challenging wide distribution of motor neurons, various therapeutic approaches have been developed in order to address these issues. The present chapter concentrates its scope to gene transfer technology. Our main emphasis will be on ALS, since animal models of this disease have been the most wildly used for gene therapy approaches.
