RNA-LNP-mediated in vivo prime editing corrects disease phenotypes in a mouse model of citrullinemia type I.
Talas A, Ioannidi EI, Weber Y, Haenggi T, Kulcsar PI, Zurcher N, Faccin E, Moon WJ, Kissling L, Villiger EA, Matsushita M, Rothgangl T, Janjuha S, Lin PJC, Poms M, Muramatsu H, Vadovics M, Reichmuth A, Kopf M, Pardi N, Tam YK, Haberle J, Schwank G
Sci Transl Med. 2026 May 13;18(849):eaec7274. doi: 10.1126/scitranslmed.aec7274. Epub 2026 May 13.
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Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 251104 | pAT15535_hU6_epegRNA_Ef1a-mCh | epegRNA cloning plasmid + mCherry expression |
| 251105 | pAT159_AAV_p3_PE7-C_hU6_epegRNA | Encodes the C terminal of the PE7 prime editor in an AAV expression plasmid + pegRNA cloning casette |
| 251106 | pAT172_AAV_p3_PE7-N | Encodes the N terminal of the PE7 prime editor in an AAV expression plasmid |