scholarly journals Cytotoxicity of lectins toward skin fibroblasts from patients with Duchenne muscular dystrophy and myotonic dystrophy

1986 ◽  
Vol 31 (2) ◽  
pp. 85-91 ◽  
Author(s):  
Kousaku Ohno ◽  
Chizuko Nakano ◽  
Shougo Ishii ◽  
Kenzo Takeshita
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Myotonic Dystrophy ◽  
Skin Fibroblasts

Hyperproliferation of synapses on spinal motor neurons of Duchenne muscular dystrophy and myotonic dystrophy patients

2003 ◽  
Vol 106 (6) ◽  
pp. 557-560 ◽  
Author(s):  
Masahiro Nagao ◽  
Shuichi Kato ◽  
Hideaki Hayashi ◽  
Hidemi Misawa
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Myotonic Dystrophy ◽  
Motor Neurons ◽  
Spinal Motor Neurons ◽  
Spinal Motor

scholarly journals Emerging strategies for cell and gene therapy of the muscular dystrophies

2009 ◽  
Vol 11 ◽  
Author(s):  
Lindsey A. Muir ◽  
Jeffrey S. Chamberlain
Keyword(s):  
Gene Therapy ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Myotonic Dystrophy ◽  
Trinucleotide Repeat ◽  
Chromosome 1 ◽  
Muscular Dystrophies ◽  
Dystrophic Muscle ◽  
Kinase Gene ◽  
Wide Range

The muscular dystrophies are a heterogeneous group of over 40 disorders that are characterised by muscle weakness and wasting. The most common are Duchenne muscular dystrophy and Becker muscular dystrophy, which result from mutations within the gene encoding dystrophin; myotonic dystrophy type 1, which results from an expanded trinucleotide repeat in the myotonic dystrophy protein kinase gene; and facioscapulohumeral dystrophy, which is associated with contractions in the subtelomeric region of human chromosome 1. Currently the only treatments involve clinical management of symptoms, although several promising experimental strategies are emerging. These include gene therapy using adeno-associated viral, lentiviral and adenoviral vectors and nonviral vectors, such as plasmid DNA. Exon-skipping and cell-based therapies have also shown promise in the effective treatment and regeneration of dystrophic muscle. The availability of numerous animal models for Duchenne muscular dystrophy has enabled extensive testing of a wide range of therapeutic approaches for this type of disorder. Consequently, we focus here on the therapeutic developments for Duchenne muscular dystrophy as a model of the types of approaches being considered for various types of dystrophy. We discuss the advantages and limitations of each therapeutic strategy, as well as prospects and recent successes in the context of future clinical applications.

scholarly journals Protein degradation in skin fibroblasts from patients with Duchenne muscular dystrophy

1980 ◽  
Vol 192 (1) ◽  
pp. 257-262 ◽  
Author(s):  
H E Statham ◽  
J A Witkowski ◽  
V Dubowitz
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Protein Degradation ◽  
Extracellular Space ◽  
Skin Fibroblasts ◽  
Growth State ◽  
Cell Age ◽  
Normal Controls ◽  
H 30

The rates of degradation of [3H]leucine-labelled proteins have been measured in cultures of skin fibroblasts obtained from normal controls (five subjects) and patients with Duchenne muscular dystrophy (six subjects). Cultures were incubated with [3H]leucine (10 microCi/ml) for 60 min to label “short-lived” proteins, and with [3H]leucine (5 microCi/ml) for 60 h to label “long-lived” proteins. Optimal wash procedures were devised for removal of [3H]leucine from the extracellular space and from cell pools before beginning degradation measurements. Re-utilization of [3H]leucine released from degraded labelled proteins was prevented by supplementing the medium with 4mM-leucine. Rates of degradation did not depend on the growth state of the cells or on cell age over the range used (passages eight-20). Degradation of long-lived proteins was approximately linear over a 24h period, at a rate of 1.0% per h. 30% of short-lived protein was degraded within 6h. No differences were observed between protein degradation in normal fibroblasts and in those from patients with Duchenne muscular dystrophy.

Polyacrylamide-gel-electrophoretic analysis of cultured skin fibroblasts from patients with Duchenne muscular dystrophy

1981 ◽  
Vol 9 (1) ◽  
pp. 118-119 ◽  
Author(s):  
ARTHUR H. M. BURGHES ◽  
MICHAEL J. DUNN ◽  
JAN A. WITKOWSKI ◽  
VICTOR DUBOWITZ
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Electrophoretic Analysis ◽  
Polyacrylamide Gel ◽  
Skin Fibroblasts ◽  
Cultured Skin
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