Journal article
Functional Rescue of the Sarcoglycan Complex in the BIO 14.6 Hamster Using δ-Sarcoglycan Gene Transfer
Molecular cell, Vol.1(6), pp.841-848
1998
DOI: 10.1016/S1097-2765(00)80083-0
PMID: 9660967
Abstract
Four types of limb-girdle muscular dystrophy (LGMD) are known to be caused by mutations in distinct sarcoglycan genes. The BIO 14.6 hamster is a model for sarcoglycan-deficient LGMD with a deletion in the δ-sarcoglycan (δ-SG) gene. We investigated the function of the sarcoglycan complex and the feasibility of sarcoglycan gene transfer for LGMD using a recombinant δ-SG adenovirus in the BIO 14.6 hamster. We demonstrate extensive long-term expression of δ-sarcoglycan and rescue of the entire sarcoglycan complex, as well as restored stable association of α-dystroglycan with the sarcolemma. Importantly, muscle fibers expressing δ-sarcoglycan lack morphological markers of muscular dystrophy and exhibit restored plasma membrane integrity. In summary, the sarcoglycan complex is requisite for the maintenance of sarcolemmal integrity, and primary mutations in individual sarcoglycan components can be corrected in vivo.
Details
- Title: Subtitle
- Functional Rescue of the Sarcoglycan Complex in the BIO 14.6 Hamster Using δ-Sarcoglycan Gene Transfer
- Creators
- Kathleen H Holt - Howard Hughes Medical Institute, Department of Physiology and Biophysics, and Department of Neurology, University of Iowa College of Medicine, Iowa City, Iowa 52242, USALeland E Lim - Howard Hughes Medical Institute, Department of Physiology and Biophysics, and Department of Neurology, University of Iowa College of Medicine, Iowa City, Iowa 52242, USAVolker Straub - Howard Hughes Medical Institute, Department of Physiology and Biophysics, and Department of Neurology, University of Iowa College of Medicine, Iowa City, Iowa 52242, USADavid P Venzke - Howard Hughes Medical Institute, Department of Physiology and Biophysics, and Department of Neurology, University of Iowa College of Medicine, Iowa City, Iowa 52242, USAFranck Duclos - Howard Hughes Medical Institute, Department of Physiology and Biophysics, and Department of Neurology, University of Iowa College of Medicine, Iowa City, Iowa 52242, USARichard D Anderson - Department of Internal Medicine, University of Iowa College of Medicine, Iowa City, Iowa 52242, USABeverly L Davidson - Department of Internal Medicine, University of Iowa College of Medicine, Iowa City, Iowa 52242, USAKevin P Campbell - Howard Hughes Medical Institute, Department of Physiology and Biophysics, and Department of Neurology, University of Iowa College of Medicine, Iowa City, Iowa 52242, USA
- Resource Type
- Journal article
- Publication Details
- Molecular cell, Vol.1(6), pp.841-848
- Publisher
- Elsevier Inc
- DOI
- 10.1016/S1097-2765(00)80083-0
- PMID
- 9660967
- ISSN
- 1097-2765
- eISSN
- 1097-4164
- Language
- English
- Date published
- 1998
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Iowa Neuroscience Institute
- Record Identifier
- 9984068381802771
Metrics
15 Record Views