Research press release


Nature Biotechnology

Fresh lead for broad-spectrum Huntington's therapy



D Coreyたちは、遺伝子操作したRNAを用いれば、変異HTTと正常HTTがCAG反復配列自体に基づいて識別できることを明らかにした。この方法では、ハンチントン病と関連のみられる最も一般的な変異をもたない患者の細胞でも、変異遺伝子を選択的に標的にできる。どのような機構がかかわるのか、またこの治療法が動物でどの程度うまく働くかなど、研究すべきことは多く残されているが、今回の成果は、あらゆるハンチントン病患者に効く薬を開発する出発点になるだろう。

New findings may catalyze the development of drugs to treat Huntington's and other similar diseases, reports a study published online this week in Nature Biotechnology.

We all carry two slightly different copies of most of our genes. Huntington's disease, a severe neurological disorder, results when a sequence of three bases, CAG, in one of the two copies of the huntingtin (HTT) gene is repeated more than 36 times. The challenge in finding a cure is to silence the defective "stuttering" HTT without switching off the normal copy. So far, research into silencing mutant HTT relied on targeting specific mutations outside the repeats. But these strategies would not help patients with rare HTT mutations.

David Corey and colleagues show that an engineered RNA can discriminate between mutant and normal HTT on the basis of the repeated CAG sequence itself. They selectively target the mutant gene even in patient cells without the variants most commonly associated with Huntington's. Although more remains to be learned about the mechanisms involved and how well the treatment works in animals, the findings provide a new starting point for developing drugs that may eventually benefit all Huntington's patients.

doi: 10.1038/nbt.1539


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