Research press release


Nature Biomedical Engineering

Efficient non-viral delivery of CRISPR machinery



Niren Murthyたちは、CRISPR要素を1つ1つの金ナノ粒子の周囲にまとめて保護ポリマーで覆うことができること、そしてそのナノ粒子がCRISPR要素をさまざまな種類の細胞へ効率的に送達することを明らかにした。また、相同組換え修復(DNA切断酵素Cas9が生じた二本鎖DNA切断を修復する最も正確な機構)によって遺伝子編集が行われること、そしてデュシェンヌ型筋ジストロフィー治療を行うマウスの筋組織のオフターゲット編集レベルが最小限であったことも示している。

The delivery of CRISPR genome-editing molecules via nanoparticles rather than via viruses is described in a paper published online this week in Nature Biomedical Engineering. The non-viral approach is also shown to efficiently correct the genetic mutation causing Duchenne muscular dystrophy in mice.

The potential of CRISPR technology for healthcare applications depends on the effective delivery of three DNA-editing components - the Cas9 DNA-cleaving enzyme, the guide RNA that targets the gene to be corrected, and the DNA donor to be inserted into the genome - to the specific target cells. Viruses can be used to carry these molecules, but they are associated with safety concerns, limited cargo capacity and can decrease the editing efficiency of the CRISPR components.

Niren Murthy and colleagues show that the CRISPR components can be packaged around individual gold nanoparticles and wrapped up in a protecting polymer, and that the nanoparticles deliver the CRISPR components into a wide variety of cells efficiently. The researchers also show that gene editing occurs via homology-directed repair - the most accurate mechanism for repairing the double-strand DNA breaks created by the Cas9 DNA-cleaving enzyme - and that the levels of off-target editing in muscle tissue of the mice treated for Duchenne muscular dystrophy were minimal.

doi: 10.1038/s41551-017-0137-2


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。