Déficit de alfa-1 antitripsina: fisiopatología, enfermedades relacionadas, diagnóstico y tratamiento

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1-antitripsina 1st International Meeting on Rare Respiratory Diseases Valencia Febrero 2016. 46. Yusa K, Rashid ST, Strick-Marchand H, Varela I, Liu PQ, Paschon DE, Miranda E, Ordóñez A, Hannan NR, Rouhani FJ, Darche S, Alexander G, Marciniak SJ, Fusaki N, Hasegawa M, Holmes MC, Di Santo JP, Lomas DA, Bradley A, Vallier L. Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells. Nature. 2011;478:391-4. 47. Sandhaus RA. Gene therapy meets stem cells. N Engl J Med. 2012;366:567-9. 48. Jain AB, Patil V, Sheikh B, Apostolakos M, Ryan C, Kashyap R, Orloff M. Effect of liver transplant on pulmonary functions in adult patients with alpha 1 antitrypsin deficiency: 7 cases. Exp Clin Transplant. 2010;8:4-8. 49. Blanco I, Lara B, de Serres F. Efficacy of alpha1-antitrypsin augmentation therapy in conditions other than pulmonary emphysema. Orphanet J Rare Dis. 2011;6:14. 50. Lewis EC. Expanding the clinical indications for α(1)-antitrypsin therapy. Mol Med. 2012;18:957-70. 51. Wanner A, Arce AD, Pardee E. Novel therapeutic uses of alpha-1 antitrypsin: a window to the future. COPD. 2012;9:583-8. 52. de Serres F, Blanco I. Role of alpha-1 antitrypsin in human health and disease. J Intern Med. 2014;276:311-35.

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