The frequency of revertants in mdx mouse genetic models for Duchenne muscular dystrophy

I Danko, V Chapman, JA Wolff - Pediatric research, 1992 - nature.com
I Danko, V Chapman, JA Wolff
Pediatric research, 1992nature.com
The mdx mouse has been used for the development of cellular and gene therapies for
Duchenne muscular dystrophy. The relatively frequent occurrence of dystrophin-positive
muscle cells called revertants has hampered these efforts by interfering with data
interpretation. The mdx4 cv and mdx5 cv dystrophin mouse mutants have approximately 10-
fold fewer revertants than the mdx mutant at both 2 and 6 mo. The mdx3 cv dystrophin
mouse mutant may be a useful model for some types of human dystrophin deficiencies in …
Abstract
The mdx mouse has been used for the development of cellular and gene therapies for Duchenne muscular dystrophy. The relatively frequent occurrence of dystrophin-positive muscle cells called revertants has hampered these efforts by interfering with data interpretation. The mdx4 cv and mdx5 cv dystrophin mouse mutants have approximately 10-fold fewer revertants than the mdx mutant at both 2 and 6 mo. The mdx3 cv dystrophin mouse mutant may be a useful model for some types of human dystrophin deficiencies in which the levels of dystrophin are low but not completely absent.
nature.com