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Page 1
A largely random AAV integration profile after LPLD gene therapy.
Kaeppel C, Beattie SG, Fronza R, van Logtenstein R, Salmon F, Schmidt S, Wolf S, Nowrouzi A, Glimm H, von Kalle C, Petry H, Gaudet D, Schmidt M. Kaeppel C, et al. Nat Med. 2013 Jul;19(7):889-91. doi: 10.1038/nm.3230. Epub 2013 Jun 16. Nat Med. 2013. PMID: 23770691
An unbiased genome-wide analysis of zinc-finger nuclease specificity.
Gabriel R, Lombardo A, Arens A, Miller JC, Genovese P, Kaeppel C, Nowrouzi A, Bartholomae CC, Wang J, Friedman G, Holmes MC, Gregory PD, Glimm H, Schmidt M, Naldini L, von Kalle C. Gabriel R, et al. Among authors: kaeppel c. Nat Biotechnol. 2011 Aug 7;29(9):816-23. doi: 10.1038/nbt.1948. Nat Biotechnol. 2011. PMID: 21822255
Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria.
Pañeda A, Lopez-Franco E, Kaeppel C, Unzu C, Gil-Royo AG, D'Avola D, Beattie SG, Olagüe C, Ferrero R, Sampedro A, Mauleon I, Hermening S, Salmon F, Benito A, Gavira JJ, Cornet ME, del Mar Municio M, von Kalle C, Petry H, Prieto J, Schmidt M, Fontanellas A, González-Aseguinolaza G. Pañeda A, et al. Among authors: kaeppel c. Hum Gene Ther. 2013 Dec;24(12):1007-17. doi: 10.1089/hum.2013.166. Hum Gene Ther. 2013. PMID: 24070415
Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques.
Mattar CN, Nathwani AC, Waddington SN, Dighe N, Kaeppel C, Nowrouzi A, Mcintosh J, Johana NB, Ogden B, Fisk NM, Davidoff AM, David A, Peebles D, Valentine MB, Appelt JU, von Kalle C, Schmidt M, Biswas A, Choolani M, Chan JK. Mattar CN, et al. Among authors: kaeppel c. Mol Ther. 2011 Nov;19(11):1950-60. doi: 10.1038/mt.2011.107. Epub 2011 May 31. Mol Ther. 2011. PMID: 21629224 Free PMC article.