ABSTRACT

The ultimate therapeutic goal for Huntington’s disease (HD) is to develop diseasemodifying therapies able to (1) delay or prevent clinical illness in those who are at genetic risk; and (2) slow the progression and permit some recovery in those who have manifest clinical illness. Rapidly advancing basic and translational research has identi ed numerous potential targets for neuroprotection. Some targets may be generically neuroprotective and relevant for a variety of neurological insults, whereas others may be more selective for HD. None yet stand out suf ciently to enable concentrating efforts on just a few of these, with the exception of the huntingtin protein itself, which does not have a conventional pharmacology with which to work.