Gene-Targeted Therapies for the Central Nervous System

The identification of the genes and proteins involved in many neurodegenerative diseases 1 offers the exciting possibility of modifying those disease-linked proteins to develop novel, targeted therapies for diseases such as amyotrophic lateral sclerosis (ALS) or Huntington disease. In many of these diseases, the simplest modification-decreasing the amount of the offending protein-may represent a potent therapy. This realization, coupled with great strides in the techniques for decreasing specific proteins, has set the stage for moving these new therapies from animal models to clinical trials in the near future.