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Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.
Paulk NK, Pekrun K, Zhu E, Nygaard S, Li B, Xu J, Chu K, Leborgne C, Dane AP, Haft A, Zhang Y, Zhang F, Morton C, Valentine MB, Davidoff AM, Nathwani AC, Mingozzi F, Grompe M, Alexander IE, Lisowski L, Kay MA. Paulk NK, et al. Mol Ther. 2018 Jan 3;26(1):289-303. doi: 10.1016/j.ymthe.2017.09.021. Epub 2017 Sep 25. Mol Ther. 2018. PMID: 29055620 Free PMC article.
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.
Puzzo F, Colella P, Biferi MG, Bali D, Paulk NK, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Meliani A, Cohen-Tannoudji M, Astord S, Gjata B, Sellier P, van Wittenberghe L, Vignaud A, Boisgerault F, Barkats M, Laforet P, Kay MA, Koeberl DD, Ronzitti G, Mingozzi F. Puzzo F, et al. Among authors: paulk nk. Sci Transl Med. 2017 Nov 29;9(418):eaam6375. doi: 10.1126/scitranslmed.aam6375. Sci Transl Med. 2017. PMID: 29187643 Free PMC article.
Comparison of highly pure rAAV9 vector stocks produced in suspension by PEI transfection or HSV infection reveals striking quantitative and qualitative differences.
Trivedi PD, Yu C, Chaudhuri P, Johnson EJ, Caton T, Adamson L, Byrne BJ, Paulk NK, Clément N. Trivedi PD, et al. Among authors: paulk nk. Mol Ther Methods Clin Dev. 2021 Dec 25;24:154-170. doi: 10.1016/j.omtm.2021.12.006. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2021. PMID: 35071688 Free PMC article.
Methods Matter: Standard Production Platforms for Recombinant AAV Produce Chemically and Functionally Distinct Vectors.
Rumachik NG, Malaker SA, Poweleit N, Maynard LH, Adams CM, Leib RD, Cirolia G, Thomas D, Stamnes S, Holt K, Sinn P, May AP, Paulk NK. Rumachik NG, et al. Among authors: paulk nk. Mol Ther Methods Clin Dev. 2020 May 22;18:98-118. doi: 10.1016/j.omtm.2020.05.018. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32995354 Free PMC article.
Robust expansion of human hepatocytes in Fah-/-/Rag2-/-/Il2rg-/- mice.
Azuma H, Paulk N, Ranade A, Dorrell C, Al-Dhalimy M, Ellis E, Strom S, Kay MA, Finegold M, Grompe M. Azuma H, et al. Nat Biotechnol. 2007 Aug;25(8):903-10. doi: 10.1038/nbt1326. Epub 2007 Jul 29. Nat Biotechnol. 2007. PMID: 17664939 Free PMC article.
17 results