Dystrophin expression and somatic reversion in prednisone-treated and untreated Duchenne dystrophy

K. L. Burrow, D. D. Coovert, Christopher Jon Klein, D. E. Bulman, J. T. Kissel, K. W. Rammohan, A. H M Burghes, J. R. Mendell

Research output: Contribution to journalArticle

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Abstract

The mechanism by which prednisone improves muscle strength and function in Duchenne muscular dystrophy (DMD) is unknown. We addressed the possibility that clinical improvement was related to prednisone-induced alterations in skeletal muscle dystrophin. We performed muscle biopsies on patients at the conclusion of a randomized, doubleblind, 6-month trial of prednisone and analyzed dystrophin content using Western blots and antibody staining of tissue sections. These studies demonstrated no significant differences in dystrophin content between treatment (prednisone 1.5 mg/kg/d, n = 12; prednisone 0.75 mg/kg/d, n = 9) and placebo (n = 12) groups. Of interest, however, was the presence of varying numbers of dystrophin-positive fibers (revertants) occurring individually or in clusters in antibody-stained tissue sections of more than one-half of the Duchenne patients. Mutation analysis revealed that revertants occurred in DMD patients with identifiable deletions or duplications, and in nondeletion patients. Prednisone treatment did not influence the prevalence of revertants. Revertants are most likely due to a second-site mutation occurring in a somatic cell allowing for restoration of the translational reading frame of the dystrophin transcript.

Original languageEnglish (US)
Pages (from-to)661-666
Number of pages6
JournalNeurology
Volume41
Issue number5
StatePublished - May 1991
Externally publishedYes

Fingerprint

Dystrophin
Prednisone
Duchenne Muscular Dystrophy
Reading Frames
Mutation
Antibodies
Muscle Strength
Skeletal Muscle
Western Blotting
Placebos
Staining and Labeling
Biopsy
Muscles
Therapeutics

ASJC Scopus subject areas

  • Neuroscience(all)

Cite this

Burrow, K. L., Coovert, D. D., Klein, C. J., Bulman, D. E., Kissel, J. T., Rammohan, K. W., ... Mendell, J. R. (1991). Dystrophin expression and somatic reversion in prednisone-treated and untreated Duchenne dystrophy. Neurology, 41(5), 661-666.

Dystrophin expression and somatic reversion in prednisone-treated and untreated Duchenne dystrophy. / Burrow, K. L.; Coovert, D. D.; Klein, Christopher Jon; Bulman, D. E.; Kissel, J. T.; Rammohan, K. W.; Burghes, A. H M; Mendell, J. R.

In: Neurology, Vol. 41, No. 5, 05.1991, p. 661-666.

Research output: Contribution to journalArticle

Burrow, KL, Coovert, DD, Klein, CJ, Bulman, DE, Kissel, JT, Rammohan, KW, Burghes, AHM & Mendell, JR 1991, 'Dystrophin expression and somatic reversion in prednisone-treated and untreated Duchenne dystrophy', Neurology, vol. 41, no. 5, pp. 661-666.
Burrow KL, Coovert DD, Klein CJ, Bulman DE, Kissel JT, Rammohan KW et al. Dystrophin expression and somatic reversion in prednisone-treated and untreated Duchenne dystrophy. Neurology. 1991 May;41(5):661-666.
Burrow, K. L. ; Coovert, D. D. ; Klein, Christopher Jon ; Bulman, D. E. ; Kissel, J. T. ; Rammohan, K. W. ; Burghes, A. H M ; Mendell, J. R. / Dystrophin expression and somatic reversion in prednisone-treated and untreated Duchenne dystrophy. In: Neurology. 1991 ; Vol. 41, No. 5. pp. 661-666.
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