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Gee, Peter; Lung, Mandy S Y; Okuzaki, Yuya; Sasakawa, Noriko; Iguchi, Takahiro; Makita, Yukimasa; Hozumi, Hiroyuki; Miura, Yasutomo; Yang, Lucy F; Iwasaki, Mio; Wang, Xiou H; Waller, Matthew A; Shirai, Nanako; Abe, Yasuko O; Fujita, Yoko; Watanabe, Kei; Kagita, Akihiro; Iwabuchi, Kumiko A; Yasuda, Masahiko; Xu, Huaigeng; Noda, Takeshi; Komano, Jun; Sakurai, Hidetoshi; Inukai, Naoto; Hotta, Akitsu
Nature communications, 03/2020, Letnik: 11, Številka: 1Journal Article
Prolonged expression of the CRISPR-Cas9 nuclease and gRNA from viral vectors may cause off-target mutagenesis and immunogenicity. Thus, a transient delivery system is needed for therapeutic genome editing applications. Here, we develop an extracellular nanovesicle-based ribonucleoprotein delivery system named NanoMEDIC by utilizing two distinct homing mechanisms. Chemical induced dimerization recruits Cas9 protein into extracellular nanovesicles, and then a viral RNA packaging signal and two self-cleaving riboswitches tether and release sgRNA into nanovesicles. We demonstrate efficient genome editing in various hard-to-transfect cell types, including human induced pluripotent stem (iPS) cells, neurons, and myoblasts. NanoMEDIC also achieves over 90% exon skipping efficiencies in skeletal muscle cells derived from Duchenne muscular dystrophy (DMD) patient iPS cells. Finally, single intramuscular injection of NanoMEDIC induces permanent genomic exon skipping in a luciferase reporter mouse and in mdx mice, indicating its utility for in vivo genome editing therapy of DMD and beyond.
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Dostop do baze podatkov JCR je dovoljen samo uporabnikom iz Slovenije. Vaš trenutni IP-naslov ni na seznamu dovoljenih za dostop, zato je potrebna avtentikacija z ustreznim računom AAI.
Leto | Faktor vpliva | Izdaja | Kategorija | Razvrstitev | ||||
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JCR | SNIP | JCR | SNIP | JCR | SNIP | JCR | SNIP |
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Povezave do osebnih bibliografij avtorjev | Povezave do podatkov o raziskovalcih v sistemu SICRIS |
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Vir: Osebne bibliografije
in: SICRIS
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