Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping

Expression of Cas9 and gRNA from viral vectors in vivo may cause off-target activity. Here the authors present NanoMEDIC, which uses nanovesicles to transiently deliver editing machinery to hard-to-transfect cells.

Guardado en:
Detalles Bibliográficos
Autores principales: Peter Gee, Mandy S. Y. Lung, Yuya Okuzaki, Noriko Sasakawa, Takahiro Iguchi, Yukimasa Makita, Hiroyuki Hozumi, Yasutomo Miura, Lucy F. Yang, Mio Iwasaki, Xiou H. Wang, Matthew A. Waller, Nanako Shirai, Yasuko O. Abe, Yoko Fujita, Kei Watanabe, Akihiro Kagita, Kumiko A. Iwabuchi, Masahiko Yasuda, Huaigeng Xu, Takeshi Noda, Jun Komano, Hidetoshi Sakurai, Naoto Inukai, Akitsu Hotta
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2020
Materias:
Q
Acceso en línea:https://doaj.org/article/4d0fe2ef9a604f0fb6865d1e08b1ed50
Etiquetas: Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!
id oai:doaj.org-article:4d0fe2ef9a604f0fb6865d1e08b1ed50
record_format dspace
spelling oai:doaj.org-article:4d0fe2ef9a604f0fb6865d1e08b1ed502021-12-02T13:31:14ZExtracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping10.1038/s41467-020-14957-y2041-1723https://doaj.org/article/4d0fe2ef9a604f0fb6865d1e08b1ed502020-03-01T00:00:00Zhttps://doi.org/10.1038/s41467-020-14957-yhttps://doaj.org/toc/2041-1723Expression of Cas9 and gRNA from viral vectors in vivo may cause off-target activity. Here the authors present NanoMEDIC, which uses nanovesicles to transiently deliver editing machinery to hard-to-transfect cells.Peter GeeMandy S. Y. LungYuya OkuzakiNoriko SasakawaTakahiro IguchiYukimasa MakitaHiroyuki HozumiYasutomo MiuraLucy F. YangMio IwasakiXiou H. WangMatthew A. WallerNanako ShiraiYasuko O. AbeYoko FujitaKei WatanabeAkihiro KagitaKumiko A. IwabuchiMasahiko YasudaHuaigeng XuTakeshi NodaJun KomanoHidetoshi SakuraiNaoto InukaiAkitsu HottaNature PortfolioarticleScienceQENNature Communications, Vol 11, Iss 1, Pp 1-18 (2020)
institution DOAJ
collection DOAJ
language EN
topic Science
Q
spellingShingle Science
Q
Peter Gee
Mandy S. Y. Lung
Yuya Okuzaki
Noriko Sasakawa
Takahiro Iguchi
Yukimasa Makita
Hiroyuki Hozumi
Yasutomo Miura
Lucy F. Yang
Mio Iwasaki
Xiou H. Wang
Matthew A. Waller
Nanako Shirai
Yasuko O. Abe
Yoko Fujita
Kei Watanabe
Akihiro Kagita
Kumiko A. Iwabuchi
Masahiko Yasuda
Huaigeng Xu
Takeshi Noda
Jun Komano
Hidetoshi Sakurai
Naoto Inukai
Akitsu Hotta
Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping
description Expression of Cas9 and gRNA from viral vectors in vivo may cause off-target activity. Here the authors present NanoMEDIC, which uses nanovesicles to transiently deliver editing machinery to hard-to-transfect cells.
format article
author Peter Gee
Mandy S. Y. Lung
Yuya Okuzaki
Noriko Sasakawa
Takahiro Iguchi
Yukimasa Makita
Hiroyuki Hozumi
Yasutomo Miura
Lucy F. Yang
Mio Iwasaki
Xiou H. Wang
Matthew A. Waller
Nanako Shirai
Yasuko O. Abe
Yoko Fujita
Kei Watanabe
Akihiro Kagita
Kumiko A. Iwabuchi
Masahiko Yasuda
Huaigeng Xu
Takeshi Noda
Jun Komano
Hidetoshi Sakurai
Naoto Inukai
Akitsu Hotta
author_facet Peter Gee
Mandy S. Y. Lung
Yuya Okuzaki
Noriko Sasakawa
Takahiro Iguchi
Yukimasa Makita
Hiroyuki Hozumi
Yasutomo Miura
Lucy F. Yang
Mio Iwasaki
Xiou H. Wang
Matthew A. Waller
Nanako Shirai
Yasuko O. Abe
Yoko Fujita
Kei Watanabe
Akihiro Kagita
Kumiko A. Iwabuchi
Masahiko Yasuda
Huaigeng Xu
Takeshi Noda
Jun Komano
Hidetoshi Sakurai
Naoto Inukai
Akitsu Hotta
author_sort Peter Gee
title Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping
title_short Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping
title_full Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping
title_fullStr Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping
title_full_unstemmed Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping
title_sort extracellular nanovesicles for packaging of crispr-cas9 protein and sgrna to induce therapeutic exon skipping
publisher Nature Portfolio
publishDate 2020
url https://doaj.org/article/4d0fe2ef9a604f0fb6865d1e08b1ed50
work_keys_str_mv AT petergee extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT mandysylung extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT yuyaokuzaki extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT norikosasakawa extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT takahiroiguchi extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT yukimasamakita extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT hiroyukihozumi extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT yasutomomiura extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT lucyfyang extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT mioiwasaki extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT xiouhwang extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT matthewawaller extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT nanakoshirai extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT yasukooabe extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT yokofujita extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT keiwatanabe extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT akihirokagita extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT kumikoaiwabuchi extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT masahikoyasuda extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT huaigengxu extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT takeshinoda extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT junkomano extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT hidetoshisakurai extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT naotoinukai extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
AT akitsuhotta extracellularnanovesiclesforpackagingofcrisprcas9proteinandsgrnatoinducetherapeuticexonskipping
_version_ 1718392850673041408