Research press release


Nature Medicine

Gene therapy for AIDS


エイズの治療法として、遺伝子治療は魅力的な選択肢である。1回だけの処置でウイルス量を減らし、免疫系を維持し、生涯にわたる抗レトロウイルス療法を行わなくてすむ可能性があるからである。R Mitsuyasuたちは、成人のHIV感染者74人を対象にした遺伝子導入の初めての臨床試験として、二重盲検法によるランダム化プラセボ対照比較試験を行った。



The results from the first clinical trial of gene therapy in patients infected with HIV are reported online in Nature Medicine this week.

Gene therapy is an appealing option to treat AIDS, as it has the potential to be a once-only treatment that reduces viral load, preserves the immune system and avoids lifetime antiretroviral therapy. Ronald Mitsuyasu and colleagues have now completed the first randomized, double-blind, placebo-controlled, gene transfer clinical trial in 74 HIV-infected adults.

The patients received either placebo or blood stem cells carrying a molecule called OZ1, which prevented viral replication by targeting two key HIV proteins. OZ1 was safe, causing no adverse effects over the course of the trial. Although there was no statistical difference in viral load between the OZ1 and placebo group at weeks 47 and 48 of the trial, counts of CD4+ lymphocytes?the cell population that is depleted by HIV?were higher in the OZ1 group at 100 weeks.

This study indicates that gene therapy is safe and active in people with HIV and can be developed as a conventional therapy against AIDS.

Author contact:

Ronald Mitsuyasu (University of California, Los Angeles, CA, USA)

Tel: +1 310 557 1891; E-mail:

doi: 10.1038/nm.1932


メールマガジンリストの「Nature 関連誌今週のハイライト」にチェックをいれていただきますと、毎週各ジャーナルからの最新の「注目のハイライト」をまとめて皆様にお届けいたします。