臨床検査52巻13号

2008年12月発行

今月の主題 凝固制御 トピックス

血友病の遺伝子治療

著者： 窓岩清治¹²

所属機関： ¹自治医科大学医学部分子病態治療研究センター分子病態研究部 ²自治医科大学附属病院血液内科

ページ範囲：P.1603 - P.1607

1．はじめに

　遺伝子治療の究極的な目標は，欠損あるいは変異した遺伝子配列を正常配列に戻すことにより生涯にわたり病気を除去することである(図)¹⁾．血友病は，凝固因子の定量により治療効果が客観的に評価できること，有効性が発揮される閾値が比較的低いことすなわち正常の数%程度で長期発現ができるならば出血症状が劇的に改善できることなど，遺伝子治療に適した疾患といえる．本稿では，血友病に対する遺伝子治療の現状と今後の展開について概説する．

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