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Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mice with subtle antibody response.
Nat Commun. 2022 Nov 25;13(1):7275. doi: 10.1038/s41467-022-34898-y.
Nat Commun. 2022.
PMID: 36434000
Free PMC article.
Evolving AAV-delivered therapeutics towards ultimate cures.
He X, Urip BA, Zhang Z, Ngan CC, Feng B.
He X, et al. Among authors: urip ba.
J Mol Med (Berl). 2021 May;99(5):593-617. doi: 10.1007/s00109-020-02034-2. Epub 2021 Feb 16.
J Mol Med (Berl). 2021.
PMID: 33594520
Free PMC article.
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