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Pleiades Promoter Project
Gene therapy is the most promising new frontier in medical therapeutic
intervention. Worldwide, numerous gene therapy trials have been
conducted, some with devastating consequences, but some with tantalizing
successes.
From these experiences, several themes to increase safety have
emerged: 1) more clinically relevant basic research is needed;
2) promoters should have specificity to reduce inappropriate expression;
and 3) random insertion into the genome is undesirable due to possible
mutagenesis or insertion-site effects. The Pleiades Promoter Project
will address all three. The end goal of this project is to generate
160 fully characterized, human DNA MiniPromoters (less than 4 kb)
to drive gene expression in defined brain regions of therapeutic
interest for diseases such as Alzheimer, Parkinson, Huntington,
Amyotrophic Lateral Sclerosis, Multiple Sclerosis, Spinocerebellar
Ataxia, Depression, Autism, and Cancer. Intermediate deliverables
include 205 embryonic stem cell lines carrying human Maxi (contained
in BACs) or Mini (contained in plasmids) Promoters designed to
be modified for delivery of gene products in the mouse brain; 205
mouse strains expressing EGFP or EGFPcre recombinase in the brain;
45 fully characterized human DNA MaxiPromoters; and novel software
for MiniPromoter design and construction. Seventeen brain regions
of therapeutic interest have been isolated from mouse by laser
capture microscopy (LCM) and libraries have been prepared by using,
for the first time, a combination of Serial Analysis of Gene Expression
(SAGE)-Lite and LongSAGE technologies.
Project Website: http://pleiades.org/
Contact:
Russell F.Watkins, Ph.D.
Project Manager, Pleiades Promoter Project Centre for Molecular
Medicine and Therapeutics 950 West 28th Avenue Vancouver, B.C.
Canada
V5Z 4H4
Tel: (604) 875-2000 x 7100
Fax: (604) 875-3819
Email: rwatkins@cmmt.ubc.ca
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