Introduction: Many nervous system disorders are minimally responsive to existing treatments
but they are potential candidates for gene therapy, an approach that can correct the genetic …

Gene therapy for rare monogenetic neurological disorders is reaching clinics and offering
hope to families affected by these diseases. There is also potential for gene therapy to offer …

Diseases of the nervous system have devastating effects and are widely distributed among
the population, being especially prevalent in the elderly. These diseases are often caused …

Gene therapy is, potentially, a powerful tool for treating neurodegenerative diseases such as
amyotrophic lateral sclerosis (ALS), spinal muscular atrophy, Parkinson's disease (PD) and …

Gene therapy has generated enormous scientific, medical and public interest over the last
decade. Clinical trials involving approximately 2000 patients worldwide have targeted …

The use of gene therapy to target the central nervous system presents specific technical and
biological challenges. These may be overcome by using novel gene vector delivery …

Significant advances have been made in the last 20 years in understanding the basic
biology of the normal nervous system and in elucidating molecular and cellular mechanisms …

Gene therapy is rapidly emerging as a powerful therapeutic strategy for a wide range of
neurodegenerative disorders, including Alzheimer's disease (AD), Parkinson's disease (PD) …

Advances in targeted regulation of gene expression allowed new therapeutic approaches
for monogenic neurological diseases. Molecular diagnosis has paved the way to …

The complexity of the central nervous system (CNS) exposes it to a number of different
diseases, often caused by only small variations in gene sequence or expression level …