Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice.
Reichart D, Newby GA, Wakimoto H, Lun M, Gorham JM, Curran JJ, Raguram A, DeLaughter DM, Conner DA, Marsiglia JDC, Kohli S, Chmatal L, Page DC, Zabaleta N, Vandenberghe L, Liu DR, Seidman JG, Seidman C
Nat Med. 2023 Feb;29(2):412-421. doi: 10.1038/s41591-022-02190-7. Epub 2023 Feb 16.
(Link opens in a new window)
PubMed
(Link opens in a new window)
Article
Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 194651 | pABE8e-NG-403Q-Nterm-AAV | N-terminal AAV genome plasmid encoding ABE8e + sgRNA to correct the R403Q mutation in mice |
| 194652 | pABE8e-NG-403Q-Cterm-AAV | C-terminal AAV genome plasmid encoding ABE8e + sgRNA to correct the R403Q mutation in mice |