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TY - JOUR AU - Cox, Gregory A. AU - Sunada, Yoshihide AU - Campbell, Kevin P. AU - Chamberlain, Jeffrey S. PY - 1994 DA - 1994/12/01 TI - Dp71 can restore the dystrophin-associated glycoprotein complex in muscle but fails to prevent dystrophy JO - Nature Genetics SP - 333 EP - 339 VL - 8 IS - 4 AB - Two lines of transgenic mdx mice have been generated that express a 71 kD non-muscle isoform of dystrophin (Dp71) in skeletal muscle. This isoform contains the cysteine-rich and Oterminal domains of dystrophin, but lacks the N-terminal actin-binding and central spectrin-like repeat domains. Dp71 was associated with the sarcolemma membrane, where it restored normal expression and localization of all members of the dystrophin-associated glycoprotein complex. However, the skeletal muscle pathology of the transgenic mdx mice remained severe. These results indicate that the dystrophin C terminus cannot function independently to prevent dystrophic symptoms and confirms predictions based on patient data that both the N and C-terminal domains are required for normal dystrophin function. SN - 1546-1718 UR - https://doi.org/10.1038/ng1294-333 DO - 10.1038/ng1294-333 ID - Cox1994 ER -