Seattle - Fred Hutchinson Cancer Research Center

Stephen Tapscott, M.D., Ph.D.
(RG) Myotonic dystrophy (MMD) and chromatin
$ 52,852.00	7/1/98 - 6/30/00	Year 3

Summary: The objective of this study is to understand how the expansion of the unstable DNA, CTG repeat causes MMD.

Seattle - University of Washington

Craig Bennett, Ph.D.
(DG) Genetic analysis in juvenile amyotrophic lateral sclerosis (ALS4)
$ 35,000.00	1/1/00 - 12/31/00	Year 1

Summary: The goal of this work is to identify the abnormal gene on chromosome 9 which causes an adolescent-onset form of Lou Gehrig's disease.

Steven S. Carlson, Ph.D.
(RG) Neurexin as a postsynaptic organizer at the neuromuscular junction
$ 56,160.00	7/1/99 - 6/30/00	Year 1
$ 56,160.00	7/1/00 - 6/30/01	Year 2
$ 56,160.00	7/1/01 - 6/30/02	Year 3

Summary: Researchers will test the hypothesis that the newly discovered protein, neurexin is critical for clustering of synaptic receptors on muscle membranes critical to nerve-muscle function.

William A. Catterall, Ph.D.
(RG) Regulation of muscle calcium channel 
by protein phosphorylation
$ 65,989.00	7/1/99 - 6/30/00	Year 1
$ 60,000.00	7/1/00 - 6/30/01	Year 2
$ 60,000.00	7/1/01 - 6/30/02	Year 3

Summary: Forceful contractions of human muscles are caused by high frequency discharges of the motor nerves which innervate them. This project will examine the molecular mechanism of this process by analyzing the regulation of the calcium channels of normal and dystrophic skeletal muscle by rapid electrical stimulation.

Stephen Hauschka, Ph.D.
(RG) Regulatory cassettes for muscle gene therapy
$ 88,992.00	1/1/00 - 12/31/00	Year 2
$ 91,662.00	1/1/01 - 12/31/01	Year 3

Summary: Muscle gene therapy requires the control of high expression of the specific gene in muscle while only low expression in nonmuscle. Gene cassettes from these studies will be tested with all vector types used for gene transfer.

Neil Nathanson, Ph.D.
(RG) Neurokine regulation of choline 
acetyltransferase gene expression
$ 48,008.00	1/1/00 - 12/31/00	Year 3

Summary: Leukemia inhibitory factor (LIF) and ciliary neurotrophic factor (CNTF) are related proteins, which can promote the survival of motor neurons and induce the expression of choline acetyltransferase (ChAT), the enzyme which makes the neurotransmitter acetylcholine. This proposal will determine the basis for the induction by LIF and CNTF of the expression of the ChAT gene in neuronal cells.

Ruth Westenbroek, Ph.D.
(RG) Expression and localization of Ca2+ channels in spinal motor neurons
$ 46,844.00	1/1/00 - 12/31/00	Year 2
$ 48,792.00	1/1/01 - 12/31/01	Year 3

Summary: Localization and expression of calcium channels in motor neurons will be studied to further elucidate their role in the disease process of several neuromuscular disorders.

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