Resumes
Resumes
Roger Gaedigk Phones & Addresses
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7340 Park St, Shawnee, KS 66216 (913) 232-0745
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Shawnee Mission, KS
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Gresham, OR
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Bard, CA
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Shawnee Msn, KS
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7340 Park St, Shawnee, KS 66216 (913) 962-4395
Publications
Us Patents
Retinal Dystrophin Transgene And Methods Of Use Thereof
View pageUS Patent:
20080044393, Feb 21, 2008
Filed:
Feb 4, 2005
Appl. No.:
11/050911
Inventors:
Robert White - Shawnee KS, US
Roger Gaedigk - Shawnee KS, US
Roger Gaedigk - Shawnee KS, US
International Classification:
A61K 31/70
A01K 67/00
A61K 35/00
A61P 43/00
C07H 21/04
C12N 15/00
C12N 5/06
A01K 67/00
A61K 35/00
A61P 43/00
C07H 21/04
C12N 15/00
C12N 5/06
US Classification:
424093210, 435320100, 435325000, 435372000, 514044000, 536023500, 800013000
Abstract:
Duchenne muscular dystrophy (DMD) is a progressive muscle disease that is caused by severe defects in the dystrophin gene and results in the patient's death by the third decade. The present invention utilizes the Double Mutant mice (DM) as an appropriate human model for DMD as these mice are deficient for both dystrophin and utrophin (mdx/+, utrn −/−), die at 3 months of age and suffer from severe muscle weakness, pronounced growth retardation, kyphosis, weight loss, slack posture, and immobility. Expression from a transgene of novel human retinal dystrophin Dp260 was shown to prevent premature death and reduce the severe muscular dystrophy phenotype to a mild clinical myopathy. Electromyography, histology, radiography, magnetic resonance imaging, and behavior studies concluded that DM transgenic mice grew normally, had normal spinal curvature and mobility, and had reduced muscle pathology. EMG and histologic data from transgenic DM mice showed decreased abnormalities to levels typical of mild myopathy, while the DM mice exhibited severe abnormalities commonly seen in human dystrophinopathies. The transgenic DM mice also had measurable movement levels comparable to those of untreated mdx mice and controls.