High Throughput Drug Screening in Muscular Dystrophy
Application Abstract
This proposal is a request for partial financial support for a workshop
on High Throughput Drug Screening in Muscular Dystrophy to be held in
Washington DC . The meeting is proposed for 1.5 days, in the time window
between April 8-22. This workshop will be attended by representatives from
virtually all academic and pharmaceutical laboratories pursuing HTS for
muscular dystrophy, as well as key presentations on newly emerging assays
and screening technologies that have not yet been applied to muscular
dystrophy. The growing interest in HTS applied to muscular dystrophy in
both academic and pharmaceutical labs, and the lack of any previous meeting
devoted to this topic, makes this proposed workshop highly timely. The
workshop will be organized into four sessions: Session 1 will focus on
assay development (e.g. utrophin up-regulation, membrane stabilization,
protease inhibition). Session 2 will focus on drug screening facilities,
methods and drug libraries. Session 3 will focus on animal model facilities
available for pre-clinical studies of HTS hits. Session 4 will focus on
review and approval of specific deliverables by the attendees. The workshop
will be limited to 15 speakers and 10 stake holders, however we will provide
Webex transmission of the conference, with option of limited remote
questions. Deliverables include development of a web site with linked
information on speakers and slide presentations used during the workshop.
In addition, we will develop an internet accessible description of existing
muscular dystrophy HTS assay descriptions and site of development and
research on these, as well as HTS drug screening facilities with
descriptions of compound libraries and method of access.