Gene-targeted therapies for the central nervous system
TM Miller, RA Smith, H Kordasiewicz… - Archives of …, 2008 - jamanetwork.com
The identification of the genes and proteins involved in many neurodegenerative dis-eases1
offers the exciting possibility of modifying those disease-linked proteins to de-velop 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 …
offers the exciting possibility of modifying those disease-linked proteins to de-velop 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 …