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Conditional disruption of hepatic carbamoyl phosphate synthetase 1 in mice results in hyperammonemia without orotic aciduria and can be corrected by liver-directed gene therapy.
Khoja S, Nitzahn M, Hermann K, Truong B, Borzone R, Willis B, Rudd M, Palmer DJ, Ng P, Brunetti-Pierri N, Lipshutz GS. Khoja S, et al. Among authors: willis b. Mol Genet Metab. 2018 Aug;124(4):243-253. doi: 10.1016/j.ymgme.2018.04.001. Epub 2018 Apr 12. Mol Genet Metab. 2018. PMID: 29801986 Free PMC article.
Efficient mouse genome engineering by CRISPR-EZ technology.
Modzelewski AJ, Chen S, Willis BJ, Lloyd KCK, Wood JA, He L. Modzelewski AJ, et al. Nat Protoc. 2018 Jun;13(6):1253-1274. doi: 10.1038/nprot.2018.012. Epub 2018 May 10. Nat Protoc. 2018. PMID: 29748649 Free PMC article.
602 results