chapter  8
28 Pages

Chapter Pharmaceutical Development for Huntington’s Disease

Introduction ............................................................................................................ 198 People with HD Have Few Therapeutic Options .......................................... 198 The Landscape Is Favorable for HD Drug Discovery ..................................200 Cell-Based Models Suggest New Therapeutic Targets .................................200 In Vivo Models of Mutant Htt Action Are Available in Rats, Mice,

Worms, and Flies ..............................................................................200 Htt-Expressing Cells Allow High-Throughput “Phenotypic Screening” ..... 201 What Is the Function of Wild-Type Htt?.......................................................202

Mutant Htt Affects Some Cells More Than Others, but We Do Not Know Which Affected Cells Cause HD Pathogenesis ............................................202 “Phenotypic” Cell-Based Screens Have Identi ed Molecules That