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Research Grants

ACTIVE GRANTS, 1999

CALIFORNIA

FUNDING PROGRAM LEGEND
(EMG) (GRG) (INT) (RF) (RG) (SG) (DG)	Earmarked Gift Genetics Research Grant Interim Grant Research Fellowship Research Grant Special Grant Research Development Grant

Berkeley - University of California

David R. Hsu, Ph.D.
(DG)  The roles of BMP antagonists in embryonic myogenesis
$35,000.00  1/1/00 - 12/31/00	Year 2
$35,000.00  1/1/01 - 12/31/01	Year 3

Summary: Researchers will generate a mutation in the "gremlin" gene to study the effect on embryonic development in mice.

Dorit Parnas, Ph.D.
(DG)  Genes regulating synaptic structure and 
muscle integrity in Drosophila
$35,000.00  1/1/00 - 12/31/00	Year 1
$35,000.00  1/1/01 - 12/31/01	Year 2
$35,000.00  1/1/02 - 12/31/02	Year 3

Summary: Researchers isolated genes that control the neuromuscular junction structure in fruit flies. Mutations in these genes also lead to muscle degeneration and one gene may be related to dystrophin in humans.

Davis - University of California

Ricardo Maselli, M.D.
(RG)  Pathogenesis and treatment of presynaptic 
congenital myasthenia
$70,000.00  1/1/00-12/31/00	 Year 1

Summary: Objectives are to develop effective forms of diagnosis and treatment for children affected with congenital diseases that impair the transmission of electrical impulses between nerve and muscle.

Irvine - University of California

Katumi Sumikawa, Ph.D.
(RG)  Calcium entry through acetylcholine receptors 
(AChR) and its role in muscle disorders
$51,023.00  1/1/00 - 12/31/00	Year 3

Summary: Researchers seek to understand the control mechanisms for calcium entry through muscle AChRs. Altered control mechanisms may have the serious consequences preventing normal muscle function.

Luis P. Villarreal, Ph.D.
(RG)  Adenovirus vectors for genetic treatment of 
muscular dystrophies
$60,000.00  7/1/99 - 6/30/00	Year 3

Summary: Researchers will develop a new virus for use as a possible gene therapy vector for Duchenne and other muscular dystrophies.

Sara T. Winokur, Ph.D.
(RG)  Pathogenesis of FSHD: Gene expression 
profiling by microarray analysis
$139,323.00  7/1/99 - 6/30/00	Year 1

Summary: Researchers will apply the recent technology of DNA microarrays (chips) to identify the biochemical and cellular basis of facioscapulohumeral muscular dystrophy (FSHD). The GeneChip approach will generate a pathophysiologic "profile" of the disease, which can also then be used to evaluate therapeutic approaches to the disease.

La Jolla - Ludwig Institute for Cancer Research

Don Cleveland, Ph.D.
(RG)  Aberrant transport in ALS-linked SOD1 mutant mediated disease
$50,540.00  1/1/00 - 12/31/00	Year 3

Summary: In mice that are genetic mimics of inherited ALS, researchers are examining how damage to the systems that transport components along the nerves contributes to the earliest stages of disease, with an eye to identifying ways to slow disease onset or progression.

La Jolla - Salk Institute for Biological Studies

Robert Gellibolian, Ph.D.
(DG)  Molecular mechanism of CTG instability in myotonic dystrophy (MMD)
$35,000.00  7/1/99 - 6/30/00	Year 2
$35,000.00  7/1/00 - 6/30/01	Year 3

Summary: This proposal is aimed at understanding the roles of replication fork polarity, nucleosome structure, and p53 on CTG repeat expansion in the DMPK gene responsible for MMD. To date, the interrelation between chromatin structure, sequence composition, and origin of replication, remain unknown. Researchers have devised a method which will allow the study of all of these issues for the first time in one system in mammalian cells.

Daniel E. Syroid, Ph.D.
(DG)  Control of Schwann cell viability in neuromuscular disease
$35,000.00  7/1/99 - 6/30/00	Year 1
$35,000.00  7/1/00 - 6/30/01	Year 2

Summary: Schwann cells are essential for proper nerve and muscle function and repair. Researchers will attempt to control Schwann cell function which may provide strategies to prevent or treat neuromuscular disease.

La Jolla - University of California

Paul Martin, Ph.D.
(RG)  Therapies for muscular dystrophy involving a 
synaptic carbohydrate
$54,000.00  7/1/99 - 6/30/00	Year 3

Summary: This study will determine if manipulation of the carbohydrate composition of the muscle membrane can be used as a therapy for Duchenne or congenital muscular dystrophy.

Los Angeles - University of California

Michael Graves, M.D.
(EMG)  Restricted funds for ALS research
$12,500.00  4/1/99 - 3/31/00	Year 1

Carla Koehler, Ph.D.
(RG)  Understanding protein import and the link to 
mitochondrial myopathy
$70,000.00  1/1/00 - 12/31/00	Year 1
$70,000.00  1/1/01 - 12/31/01	Year 2
$70,000.00  1/1/02 - 12/31/02	Year 3

Summary: The objective is to determine how proteins assemble in the mitochondrion, the energy-producing unit of the cell with the goal of understanding the basis of mitochondrial myopathies and developing effective treatments.

Los Angeles - University of Southern California

Valerie Askanas, M.D., Ph.D.
(RG)  Pathogenesis of inclusion-body myositis (IBM)
$70,295.00  1/1/00 - 12/31/00	Year 3

Summary: Utilizing cultured normal human muscle after beta-amyloid precursor protein gene transfer and cultured hereditary IBM muscle, researchers will study oxidative stress in them. The results may shed new light for treatment of those diseases.

W. King Engel, M.D.
(EMG)  Restricted funds for support of the 
Jerry Lewis MDA/ALS Center
$150,505.00  4/1/99 - 3/31/00	Year 3

Chien-Ping Ko, Ph.D.
(RG)  The role of perisynaptic Schwann cells at the 
neuromuscular junction
$54,847.00  7/1/99 - 6/30/00	Year 3

Summary: Researchers will study the role of a special type of cell associated with the nerve-muscle contact believed to help guide nerve endings to form proper connections with muscles.

Nathalie Leclerc, Ph.D.
(DG)  BAPP overexpression and inclusion-body myositis (IBM)
$35,000.00  7/1/99 - 6/30/00	Year 1
$35,000.00  7/1/00 - 6/30/01	Year 2
$35,000.00  7/1/01 - 6/30/02	Year 3

Summary: Sporadic and hereditary IBM are highly debilitating and progressive muscle diseases for which there is no successful treatment available. Researchers are seeking to understand mechanisms that lead to the IBMs. The results may shed new light for treatment of these diseases.

Sita Reddy, Ph.D.
(RG)  Dissection of the molecular defects in 
myotonic dystrophy (MMD)
$100,000.00  1/1/00 - 12/31/00	Year 1
$100,000.00  1/1/01 - 12/31/01	Year 2
$100,000.00  1/1/02 - 12/31/02	Year 3

Summary: Myotonic dystrophy (MMD) is the most common adult onset muscular dystrophy in humans. The mechanism causing the disease can best be studied by the development and characterization of animal models. These experiments with animal models will allow important insights into the molecular mechanism of MMD.

Pasadena - California Institute of Technology

Marianne Bronner-Fraser, Ph.D.
(RG)  Segmentation of the motor axon outgrowth
$63,600.00  1/1/00 - 12/31/00	Year 3

Summary: This proposal examines the mechanisms underlying the guidance of motor axons as they move through the rostral half of each somite. Their segmental navigation leads to the segmental organization of peripheral nerves which is, in turn, important for formation of proper connections between nerves and muscle.

Rusty Lansford, Ph.D.
(DG)  Visualizing how the Eph TRK family effects 
somitogenesis and myogenesis
$35,000.00  1/1/00 - 12/31/00	Year 2
$35,000.00  1/1/01 - 12/31/01	Year 3

Summary: Researchers will determine the earliest cell and molecular events in muscle development by directly imaging cell movements in the somites of living embryos.

San Diego - San Diego State University

Sanford Bernstein, Ph.D.
(RG)  Probing muscle function by genetic suppression of myopathies
$60,000.00	1/1/00 - 12/31/00	Year 2
$60,000.00  1/1/01 - 12/31/01	Year 3

Summary: Researchers are analyzing how modified genes can prevent muscle degeneration caused by some genetic mutation.

Greg L. Harris, Ph.D.
(RG)  Molecular genetic analysis of mutations affecting muscle
$80,266.00  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: Researchers are analyzing gene mutations in Drosophila which disrupt the function of skeletal muscles. These studies are designed to provide a starting point for the molecular analysis of skeletal muscle disorders in higher organisms.

San Francisco - California Pacific Medical Center

Robert Miller, M.D.
(EMG)  Restricted funds for support of the 
Forbes Norris MDA/ALS Center
$100,327.91  4/1/99 - 3/31/00	Year 3

(EMG)	Restricted funds for support of the MDA Clinic - Zimmerman Fund
$141,135.94  4/1/99 - 3/31/00	Year 1

San Francisco - University of California

David Bredt, M.D., Ph.D.
(RG)  Actinin-associated LIM protein and 
facioscapulohumeral (FSH) muscular dystrophy
$60,781.00  1/1/00 - 12/31/00	Year 3

Summary: ALP is the first muscle-specific protein that maps in the general vicinity of the FSHD locus. This work will determine the possible role for ALP in FSHD.

Nathalie A. Compagnone, Ph.D.
(DG)  Neurosteroids and survival of motor neurons 
in model of spinal muscular atrophy (SMA)
$35,000.00  7/1/99 - 6/30/00	Year 1
$35,000.00  7/1/00 - 6/30/01	Year 2
$35,000.00  7/1/01 - 6/30/02	Year 3

Summary: Researchers will study the relationship between the SMN protein (linked to SMA) and biosynthesis of DHEA which may lead to therapeutic development.

Marc Diamond, M.D.
(DG)  Glucocorticoid-regulated modifiers of cell 
vulnerability in spinal bulbar muscular atrophy (SBMA)
$35,000.00  1/1/00 - 12/31/00	Year 1
$35,000.00  1/1/01 - 12/31/01	Year 2
$35,000.00  1/1/02 - 12/31/02	Year 3

Summary: This project will attempt to identify genes that affect cell vulnerability to the defective gene that causes SBMA, and to test potential therapies.

Yibing Li, M.D., Ph.D.
(RG)  Neuronal and glial mutant SOD1 transgenic mice for 
amyotrophic lateral sclerosis (ALS) study
$76,882.00  7/1/99 - 6/30/00	Year 2
$80,080.00  7/1/00 - 6/30/01	Year 3

Summary: This project is to study the role of neurons and glia in motoneuron degeneration in ALS through the use of transgenic mice expressing mutant SOD1 in neurons or glia.

Charles Ordahl, Ph.D.
(RG)  Myogenic progenitor cells: A new muscle stem cell class
$46,525.00  1/1/00 - 12/31/00	Year 1
$49,760.00  1/1/01 - 12/31/01	Year 2
$53,219.00  1/1/02 - 12/31/02	Year 3

Summary: One of the most potentially promising treatments for muscular dystrophy, myoblast transfer therapy, has been unsuccessful because adult myoblasts lack sufficient ability to migrate to appropriate sites, expand mitotically and then incorporate into existing muscle. A newly discovered embryonic stem cell type named myogenic progenitor cells possess many of these essential characteristics. Researchers propose to isolate and analyze such cells to determine their potential for muscle replacement therapy.

San Francisco - Veterans Administration Medical Center

Frank M. Longo, M.D., Ph.D.
(RG)  Tyrosine phosphatases: New strategies for neuropathy therapeutics
$80,000.00	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: In clinical trials, protein growth factors such as nerve growth factor have been shown to effectively treat various neuropathies. In these studies, researchers will elucidate a strategy that will allow development of medicinal compounds that will augment the effects of the body's naturally occurring growth factors.

Stanford - Stanford University

Helen Blau, Ph.D.
(RG)  Role of mutant 3 UTR in the pathogenesis of myotonic dystrophy (MMD)
$50,000.00  7/1/99 - 6/30/00	Year 3

Summary: A unique inherited molecular defect has been shown to cause MMD. Using recently developed techniques, researchers will study why this defect causes MMD. Understanding this mechanism may lead to a therapy.

Liqun Luo, Ph.D.
(RG)  Studies of fly C17 class kinases related to myotonic dystrophy (MMD)
$55,000.00  7/1/99 - 6/30/00	Year 3

Summary: A gene found in flies that helps nerve cells acquire the proper shape resembles a human gene that is defective in MMD. Researchers will study this fly gene which may help understand the disease, and possibly treat it.

Thomas A. Rando, M.D., Ph.D.
(RG)  Muscular dystrophies: Mechanisms of injury and cellular defenses
$60,000.00  1/1/00 - 12/31/00	Year 2

Summary: Researchers will study a mechanism of cellular injury that may lead to cell death in the muscular dystrophies and mechanisms by which muscle cells protect themselves against this type of injury.

(EMG)  Restricted funds for DMD research
$15,000.00  4/1/99 - 3/31/00	Year 1

Eric M. Shooter, Ph.D.
(RG)  Peripheral myelin protein 22 (PMP22) in peroneal muscular atrophy
$53,659.00  7/1/99 - 6/30/00	Year 1
$55,227.00  7/1/00 - 6/30/01	Year 2
$56,858.00  7/1/01 - 6/30/02	Year 3

Summary: In peroneal muscular atrophy, the nerves lose their sheaths and do not function well. Loss of the sheath is due to an inherited defect in one of the proteins in the cell that makes the sheath. Researchers will explore this defect and will also try to rejuvenate the nerves by giving them a protein which is known to promote their survival.

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