Duchenne muscular dystrophy: Current knowledge, treatment, and future prospects

被引:26
作者
Biggar, WD
Klamut, HJ
Demacio, PC
Stevens, DJ
Ray, PN
机构
[1] Hosp Sick Children, Res Inst, Toronto, ON M5G 1X8, Canada
[2] Hosp Sick Children, Dept Pediat Lab Med, Toronto, ON M5G 1X8, Canada
[3] Bloorview MacMillan Childrens Ctr, Toronto, ON, Canada
[4] Univ Toronto, Dept Paediat, Toronto, ON M5S 1A1, Canada
[5] Univ Toronto, Dept Med Biophys, Toronto, ON, Canada
[6] Univ Toronto, Dept Mol & Med Genet, Toronto, ON, Canada
[7] Univ Hlth Network, Ontario Canc Inst, Dept Expt Therapeut, Toronto, ON, Canada
[8] Hosp Sick Children, Dept Paediat Lab Med, Toronto, ON M5G 1X8, Canada
关键词
D O I
10.1097/01.blo.0000022197.37246.64
中图分类号
R826.8 [整形外科学]; R782.2 [口腔颌面部整形外科学]; R726.2 [小儿整形外科学]; R62 [整形外科学(修复外科学)];
学科分类号
摘要
The cloning of the dystrophin gene has led to major advances in the understanding of the molecular genetic basis of Duchenne, Becker, and other muscular dystrophies associated with mutations in genes encoding members of the dystrophin-associated glycoprotein complex. The recent introduction of pharmaceutical agents such as prednisone has shown great promise in delaying the progression of Duchenne muscular dystrophy but there remains a need to develop more longterm therapeutic interventions. Knowledge of the nature of the dystrophin gene and the glycoprotein complex has led many researchers to think that somatic gene replacement represents the most promising approach to treatment. The potential use of this strategy has been shown in the mdx mouse model of Duchenne muscular dystrophy, where germ line gene transfer of either a full-length or a smaller Becker-type dystrophin minigene prevents necrosis and restores normal muscle function.
引用
收藏
页码:88 / 106
页数:19
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