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Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria.
D'Avola D, López-Franco E, Sangro B, Pañeda A, Grossios N, Gil-Farina I, Benito A, Twisk J, Paz M, Ruiz J, Schmidt M, Petry H, Harper P, de Salamanca RE, Fontanellas A, Prieto J, González-Aseguinolaza G. D'Avola D, et al. Among authors: twisk j. J Hepatol. 2016 Oct;65(4):776-783. doi: 10.1016/j.jhep.2016.05.012. Epub 2016 May 17. J Hepatol. 2016. PMID: 27212246 Free article. Clinical Trial.
Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice.
Unzu C, Sampedro A, Mauleón I, Alegre M, Beattie SG, de Salamanca RE, Snapper J, Twisk J, Petry H, González-Aseguinolaza G, Artieda J, Rodríguez-Pena MS, Prieto J, Fontanellas A. Unzu C, et al. Among authors: twisk j. Mol Ther. 2011 Feb;19(2):243-50. doi: 10.1038/mt.2010.210. Epub 2010 Sep 28. Mol Ther. 2011. PMID: 20877347 Free PMC article.
Gene therapy for lipoprotein lipase deficiency: working toward clinical application.
Rip J, Nierman MC, Sierts JA, Petersen W, Van den Oever K, Van Raalte D, Ross CJ, Hayden MR, Bakker AC, Dijkhuizen P, Hermens WT, Twisk J, Stroes E, Kastelein JJ, Kuivenhoven JA, Meulenberg JM. Rip J, et al. Among authors: twisk j. Hum Gene Ther. 2005 Nov;16(11):1276-86. doi: 10.1089/hum.2005.16.1276. Hum Gene Ther. 2005. PMID: 16259561
Gene therapy in disorders of lipoprotein metabolism.
Vaessen SF, Twisk J, Kastelein JJ, Kuivenhoven JA. Vaessen SF, et al. Among authors: twisk j. Curr Gene Ther. 2007 Feb;7(1):35-47. doi: 10.2174/156652307779940261. Curr Gene Ther. 2007. PMID: 17305527 Review.
973 results