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Long-term effect of gene therapy on Leber's congenital amaurosis.
Bainbridge JW, Mehat MS, Sundaram V, Robbie SJ, Barker SE, Ripamonti C, Georgiadis A, Mowat FM, Beattie SG, Gardner PJ, Feathers KL, Luong VA, Yzer S, Balaggan K, Viswanathan A, de Ravel TJ, Casteels I, Holder GE, Tyler N, Fitzke FW, Weleber RG, Nardini M, Moore AT, Thompson DA, Petersen-Jones SM, Michaelides M, van den Born LI, Stockman A, Smith AJ, Rubin G, Ali RR. Bainbridge JW, et al. Among authors: beattie sg. N Engl J Med. 2015 May 14;372(20):1887-97. doi: 10.1056/NEJMoa1414221. Epub 2015 May 4. N Engl J Med. 2015. PMID: 25938638 Free PMC article. Clinical Trial.
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. Among authors: beattie sg. Nat Med. 2013 Jul;19(7):889-91. doi: 10.1038/nm.3230. Epub 2013 Jun 16. Nat Med. 2013. PMID: 23770691
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: beattie sg. 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.
Adeno-associated virus liver transduction efficiency measured by in vivo [18F]FHBG positron emission tomography imaging in rodents and nonhuman primates.
Pañeda A, Collantes M, Beattie SG, Otano I, Snapper J, Timmermans E, Guembe L, Petry H, Lanciego JL, Benito A, Prieto J, Rodriguez-Pena MS, Peñuelas I, Gonzalez-Aseguinolaza G. Pañeda A, et al. Among authors: beattie sg. Hum Gene Ther. 2011 Aug;22(8):999-1009. doi: 10.1089/hum.2010.190. Epub 2011 Apr 6. Hum Gene Ther. 2011. PMID: 21320035
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: beattie sg. Hum Gene Ther. 2013 Dec;24(12):1007-17. doi: 10.1089/hum.2013.166. Hum Gene Ther. 2013. PMID: 24070415
19 results