Progress in muscular dystrophy research with special emphasis on gene therapy
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- SUGITA Hideo
- National Center of Neurology and Psychiatry
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- TAKEDA Shin’ichi
- National Center of Neurology and Psychiatry
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Duchenne muscular dystrophy (DMD) is an X-linked, progressive muscle-wasting disease caused by mutations in the DMD gene. Since the disease was described by physicians in the 19th century, information about the subject has been accumulated. One author (Sugita) was one of the coworkers who first reported that the serum creatine kinase (CK) level is elevated in progressive muscular dystrophy patients. Even 50 years after that first report, an elevated serum CK level is still the most useful marker in the diagnosis of DMD, a sensitive index of the state of skeletal muscle, and useful to evaluate therapeutic effects. In the latter half of this article, we describe recent progress in the therapy of DMD, with an emphasis on gene therapies, particularly exon skipping.<BR><BR>(Communicated by Kunihiko SUZUKI, M.J.A.)
収録刊行物
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- Proceedings of the Japan Academy. Ser. B: Physical and Biological Sciences
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Proceedings of the Japan Academy. Ser. B: Physical and Biological Sciences 86 (7), 748-756, 2010
日本学士院
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詳細情報 詳細情報について
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- CRID
- 1390001204147458432
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- NII論文ID
- 130000309242
- 40017237080
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- NII書誌ID
- AA00785485
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- ISSN
- 13492896
- 03862208
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- NDL書誌ID
- 10779750
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- 本文言語コード
- en
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- データソース種別
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- JaLC
- NDL
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- 使用不可