Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping.
Gee P, Lung MSY, Okuzaki Y, Sasakawa N, Iguchi T, Makita Y, Hozumi H, Miura Y, Yang LF, Iwasaki M, Wang XH, Waller MA, Shirai N, Abe YO, Fujita Y, Watanabe K, Kagita A, Iwabuchi KA, Yasuda M, Xu H, Noda T, Komano J, Sakurai H, Inukai N, Hotta A
Nat Commun. 2020 Mar 13;11(1):1334. doi: 10.1038/s41467-020-14957-y.
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Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 138476 | pHLS-EF1a-FKBP12-Gag(HIV) | Expresses FKBP12 fused Gag for packaging FRB-SpCas9 into NanoMEDIC particle. |
| 138477 | pHLS-EF1a-FRB-SpCas9-A | Expresses FRB fused SpCas9 for NanoMEDIC packaging. |
| 138478 | pcDNA1-Tat | Expresses HIV Tat for efficient sgRNA packaging. |
| 138479 | pVSV-G | Expresses VSV-G envelop protein for pseudotyping NanoMEDIC particle. |
| 138480 | PL-5LTR-GW-A | For expression of ribozyme-flanked sgRNA after the Gateway (GW) LR cloning. |
| 138481 | pENTR-AmCyan | For cloning sgRNA flanked by two ribozyme elements, to subsequently cloned into PL-5LTR-GW-A vector. |
| 138482 | PL-5LTR-RGR(DMD#1)-AmCyan-A | Expresses sgRNA targeting human DMD (Dystrophin) for packaging into NanoMEDIC particle. |