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- W2321822885 abstract "先天性免疫不全症候群は，生まれもった遺伝子異常のためにある種の免疫担当細胞等に欠陥が生じ，結果として免疫能の一部あるいは広範な欠損を呈する疾患群の総称である．重篤なタイプの疾患に対して造血幹細胞を標的とした遺伝子細胞治療の研究が進められており，ADA欠損症，X連鎖重症複合免疫不全症を含むいくつかの疾患において臨床試験が行われ，臨床効果が認められている．現行の治療では，ウイルスベクターを用いた治療遺伝子のゲノムへの挿入が行われるため永続的な治療効果が望まれる反面，挿入変異とよばれる副反応による白血病の発症の危険が現実化している．本稿においては，先天性免疫不全症に対する遺伝子細胞治療の現状，抱える問題点についての概要をまとめ，現行の治療法の改善に必要な研究の必要性を私見を交えて論ずるとともに，iPS細胞を用いた治療法を含め今後期待される新たな戦略についても概説したい．" @default.
- W2321822885 created "2016-06-24" @default.
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- W2321822885 date "2010-01-01" @default.
- W2321822885 modified "2023-09-27" @default.
- W2321822885 title "Gene and Cell Therapy for Primary Immunodeficiency Diseases" @default.
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- W2321822885 doi "https://doi.org/10.2177/jsci.33.312" @default.
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