遺伝子 打開 in muscular dystrophy

Scientists have discovered how to 打ち勝つ the genetic defect that 原因（となる）s the most serious form of muscular dystrophy, it was 明らかにする／漏らすd today.

Although at an 早期に 行う／開催する／段階, the 研究 may lead to new 治療s for the 致命的な genetic 条件.

Muscular dystrophy is a rare 病気 that only 影響する／感情s boys. About one in 3,500 children in the UK are born with the most ありふれた and 厳しい form, Duchenne muscular dystrophy, each year. In the 大多数 of 事例／患者s people with the disorder die from 肺 or heart 失敗 before they are 30.

The 医療の 研究 会議 scientists (MRCs) used a technique called antisense therapy to 扱う／治療する a mouse 見解／翻訳／版 of Duchenne muscular dystrophy.

Small fragments of genetic code were used to skirt 一連の会議、交渉／完成する the 突然変異s in the dystrophin 遺伝子 that 原因（となる） the 病気.

普通は, these defects stop the 遺伝子 producing a protein that 妨げるs muscle wastage. But antisense therapy made it possible for 独房 機械／機構 that translates the genetic code to continue making the most important protein parts.

Unlike 遺伝子 therapy, antisense does not 伴う／関わる 挿入するing the 訂正するd form of the 遺伝子 into 独房s. Instead, it 'patches over' defects in the 初めの 遺伝子.

Professor Terence Partridge, from the MRCs 臨床の Sciences Centre, said: "Antisense therapy 申し込む／申し出s 抱擁する 可能性のある for the 効果的な 治療 of 病気s like Duchenne muscular dystrophy.

However, before this therapy can be 実験（する）d on humans more work must be done to 改善する the 現在の method of 配達するing the antisense 麻薬."

The 研究 was 報告（する）／憶測d in the online 版 of the 定期刊行物 Nature 薬／医学.

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