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Gene-Targeted Therapies for the Central Nervous System
Timothy M. Miller, MD, PhD;
Richard A. Smith, MD;
Holly Kordasiewicz, PhD;
Brian K. Kaspar, PhD
Arch Neurol. 2008;65(4):447-451. Published online February 11, 2008 (doi:10.1001/archneur.65.4.nnr70007).
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INTRODUCTION
The identification of the genes and proteins involved in many neurodegenerative diseases1 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.
Multiple methods are currently being studied for their ability to decrease levels of unwanted proteins, including immunization strategies, small molecules, RNA interference (RNAi), and antisense oligonucleotides. Immunization strategies were an early favorite for clearing proteins from the central nervous system (CNS), with impressive results in Alzheimer disease . . . [Full Text of this Article]
MECHANISMS OF REPRESSION
EFFICACY IN ANIMAL MODELS
CONCERNS AND CONSIDERATIONS FOR USE IN HUMANS
AUTHOR INFORMATION
Author Affiliations: Neurosciences Department, University of California, San Diego, San Diego (Dr Miller); Ludwig Institute for Cancer Research, La Jolla, California (Drs Miller and Kordasiewicz); Center for Neurologic Study, La Jolla, California (Dr Smith); and Columbus Children's Research Institute and Ohio State University, Columbus (Dr Kaspar). Dr Miller is now with the Department of Neurology, Washington University School of Medicine, St Louis, Missouri.
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