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Items: 9

1.

Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity.

Winters IP, Chiou SH, Paulk NK, McFarland CD, Lalgudi PV, Ma RK, Lisowski L, Connolly AJ, Petrov DA, Kay MA, Winslow MM.

Nat Commun. 2017 Dec 12;8(1):2053. doi: 10.1038/s41467-017-01519-y.

2.

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.

Sci Transl Med. 2017 Nov 29;9(418). pii: eaam6375. doi: 10.1126/scitranslmed.aam6375.

3.

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.

Mol Ther. 2018 Jan 3;26(1):289-303. doi: 10.1016/j.ymthe.2017.09.021. Epub 2017 Sep 25.

PMID:
29055620
4.

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

Barzel A, Paulk NK, Shi Y, Huang Y, Chu K, Zhang F, Valdmanis PN, Spector LP, Porteus MH, Gaensler KM, Kay MA.

Nature. 2015 Jan 15;517(7534):360-4. doi: 10.1038/nature13864. Epub 2014 Oct 29.

5.

In vivo selection of transplanted hepatocytes by pharmacological inhibition of fumarylacetoacetate hydrolase in wild-type mice.

Paulk NK, Wursthorn K, Haft A, Pelz C, Clarke G, Newell AH, Olson SB, Harding CO, Finegold MJ, Bateman RL, Witte JF, McClard R, Grompe M.

Mol Ther. 2012 Oct;20(10):1981-7. doi: 10.1038/mt.2012.154. Epub 2012 Aug 7.

6.

Ochronosis in a murine model of alkaptonuria is synonymous to that in the human condition.

Taylor AM, Preston AJ, Paulk NK, Sutherland H, Keenan CM, Wilson PJ, Wlodarski B, Grompe M, Ranganath LR, Gallagher JA, Jarvis JC.

Osteoarthritis Cartilage. 2012 Aug;20(8):880-6. doi: 10.1016/j.joca.2012.04.013. Epub 2012 Apr 24.

7.

AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo.

Paulk NK, Loza LM, Finegold MJ, Grompe M.

Hum Gene Ther. 2012 Jun;23(6):658-65. doi: 10.1089/hum.2012.038. Epub 2012 Jun 25.

8.

Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo.

Paulk NK, Wursthorn K, Wang Z, Finegold MJ, Kay MA, Grompe M.

Hepatology. 2010 Apr;51(4):1200-8. doi: 10.1002/hep.23481.

9.

Ploidy reductions in murine fusion-derived hepatocytes.

Duncan AW, Hickey RD, Paulk NK, Culberson AJ, Olson SB, Finegold MJ, Grompe M.

PLoS Genet. 2009 Feb;5(2):e1000385. doi: 10.1371/journal.pgen.1000385. Epub 2009 Feb 20.

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