Iowa City - University of Iowa

Valerie Allamand, Ph.D.
(DG)	Alpha-sarcoglycan gene therapy for 
limb-girdle muscular dystrophy (LGMD)
$ 35,000.00	1/1/00 - 12/31/00	Year 2

Summary: The proposed research aims at developing a viral-mediated gene transfer of a-sarcoglycan in a mouse model for LGMD2D.

Rita Barresi, Ph.D.
(DG)	Defining the molecular defect underlying 
sarcoglycan-deficient limb-girdle muscular dystrophy (LGMD)
$ 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 mechanism of sarcoglycans and related LGMD to determine possible therapeutic development.

Kevin Campbell, Ph.D.
(PPG)	Congenital muscular dystrophy (CMD) gene therapy
$ 126,257.00	1/1/00 - 12/31/00	Year 3

Summary: The goal of this research project is the development of a therapeutic strategy to treat CMD, a severe neuromuscular disease of early onset.

(EMG)	Restricted funds for limb-girdle research
$ 10,000.00	4/1/99 - 3/30/00	Year 1

Katherine Mathews, M.D.
(RG)	Characterization of myodystrophy, a possible 
mouse model for facioscapulohumeral dystrophy (FSHD)
$ 69,254.00	7/1/99 - 6/30/00	Year 2
$ 70,826.00	7/1/00 - 6/30/01	Year 3

Summary: Researchers will clarify the features of the disease and identify the gene that is abnormal in the myodystrophy mouse, a possible animal model for FSHD. This work will help us understand the basis for some forms of muscular dystrophy.

Larry Oberley, Ph.D.
(RG)	Expression of SOD1 in neuroblastoma cells: 
A novel in vitro model of amyotrophic lateral sclerosis (ALS)
$ 50,000.00	7/1/99 - 6/30/00	Year 3

Summary: Researchers will develop a novel cell culture model for the neurodegenerative disease, ALS, which will allow researchers to analyze how mutations in SOD1, the gene encoding CuZnSOD, leads to the death of motor neurons, and to identify potential therapeutic agents.

Federica Piccolo, Ph.D.
(NIDA)	Role of dysferlin in human muscular dystrophies
$ 100,000.00	1/1/00 - 12/31/00	Year 1
$ 100,000.00	1/1/01 - 12/31/01	Year 2

Summary: The goal of this project is to study a newly characterized protein called dysferlin, which is absent in a form of limb girdle muscular dystrophy and Miyoshi myopathy.

Peter Rubenstein, Ph.D.
(RG)	Control of F-actin stability and function by tropomyosin
$ 50,000.00	1/1/00 - 12/31/00	Year 3

Summary: Researchers will determine how tropomyosin stabilizes actin filaments and either alters or moves over these filaments when it regulates thin filament function during muscle contraction.

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