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Items: 1 to 20 of 29

1.

Targeted gene correction of α1-antitrypsin deficiency in induced pluripotent stem cells.

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.

Nature. 2011 Oct 12;478(7369):391-4. doi: 10.1038/nature10424.

2.

Gene correction in patient-specific iPSCs for therapy development and disease modeling.

Jang YY, Ye Z.

Hum Genet. 2016 Sep;135(9):1041-58. doi: 10.1007/s00439-016-1691-5. Epub 2016 Jun 2. Review.

PMID:
27256364
3.

Thinking outside the liver: induced pluripotent stem cells for hepatic applications.

Subba Rao M, Sasikala M, Nageshwar Reddy D.

World J Gastroenterol. 2013 Jun 14;19(22):3385-96. doi: 10.3748/wjg.v19.i22.3385. Review.

4.

New frontier in regenerative medicine: site-specific gene correction in patient-specific induced pluripotent stem cells.

Garate Z, Davis BR, Quintana-Bustamante O, Segovia JC.

Hum Gene Ther. 2013 Jun;24(6):571-83. doi: 10.1089/hum.2012.251. Review.

5.

[Recent progress of pathogenesis and treatment in alpha 1-antitrypsin deficiency].

Ozaki I, Zern MA.

Nihon Rinsho. 1999 Sep;57(9):2145-51. Review. Japanese.

PMID:
10497421
6.

Induced human pluripotent stem cells and advanced therapies: future perspectives for the treatment of haemophilia?

Liras A.

Thromb Res. 2011 Jul;128(1):8-13. doi: 10.1016/j.thromres.2011.01.010. Epub 2011 Mar 10. Review.

PMID:
21396685
7.

Generation and genetic modification of induced pluripotent stem cells.

Schambach A, Cantz T, Baum C, Cathomen T.

Expert Opin Biol Ther. 2010 Jul;10(7):1089-103. doi: 10.1517/14712598.2010.496775. Review.

PMID:
20528610
8.

[Hepatic involvement in hereditary alpha-1-antitrypsin deficiency].

Lachaux A, Dumortier J.

Rev Mal Respir. 2014 Apr;31(4):357-64. doi: 10.1016/j.rmr.2013.10.651. Epub 2014 Mar 27. Review. French.

PMID:
24750955
9.

The selective advantage of alpha1-antitrypsin deficiency.

Lomas DA.

Am J Respir Crit Care Med. 2006 May 15;173(10):1072-7. Epub 2006 Jan 26. Review.

PMID:
16439713
10.

Liver disease in alpha-1 antitrypsin deficiency: current understanding and future therapy.

Teckman JH.

COPD. 2013 Mar;10 Suppl 1:35-43. doi: 10.3109/15412555.2013.765839. Review.

PMID:
23527737
11.

Using human-induced pluripotent stem cells to model monogenic metabolic disorders of the liver.

Ordonez MP, Goldstein LS.

Semin Liver Dis. 2012 Nov;32(4):298-306. doi: 10.1055/s-0032-1329898. Epub 2013 Feb 8. Review.

PMID:
23397530
12.

α(1)-antitrypsin deficiency and inflammation.

Ekeowa UI, Marciniak SJ, Lomas DA.

Expert Rev Clin Immunol. 2011 Mar;7(2):243-52. doi: 10.1586/eci.10.95. Review.

PMID:
21426261
13.

Targeting intracellular degradation pathways for treatment of liver disease caused by α1-antitrypsin deficiency.

Wang Y, Perlmutter DH.

Pediatr Res. 2014 Jan;75(1-2):133-9. doi: 10.1038/pr.2013.190. Epub 2013 Nov 13. Review.

14.

Emerging drugs for alpha-1-antitrypsin deficiency.

Stockley RA.

Expert Opin Emerg Drugs. 2010 Dec;15(4):685-94. doi: 10.1517/14728214.2010.512287. Epub 2010 Aug 18. Review.

PMID:
20716026
15.

Homologous recombination in human iPS and ES cells for use in gene correction therapy.

Nakayama M.

Drug Discov Today. 2010 Mar;15(5-6):198-202. doi: 10.1016/j.drudis.2010.01.006. Epub 2010 Jan 29. Review.

PMID:
20116450
16.

Genetic variants of alpha1-antitrypsin.

Salahuddin P.

Curr Protein Pept Sci. 2010 Mar;11(2):101-17. Review.

PMID:
19751191
17.

α1-Antitrypsin Deficiency.

Hatipoğlu U, Stoller JK.

Clin Chest Med. 2016 Sep;37(3):487-504. doi: 10.1016/j.ccm.2016.04.011. Epub 2016 Jun 25. Review.

PMID:
27514595
18.

Alpha(1)-antitrypsin deficiency, liver disease and emphysema.

Parfrey H, Mahadeva R, Lomas DA.

Int J Biochem Cell Biol. 2003 Jul;35(7):1009-14. Review.

PMID:
12672469
19.

[From gene disease; alpha1-antitrypsine deficiency].

Hiemstra PS, Stolk J.

Ned Tijdschr Geneeskd. 2003 Apr 19;147(16):758-60. Review. Dutch.

PMID:
12731467
20.

Molecular pathogenesis of alpha-1-antitrypsin deficiency.

Duvoix A, Roussel BD, Lomas DA.

Rev Mal Respir. 2014 Dec;31(10):992-1002. doi: 10.1016/j.rmr.2014.03.015. Epub 2014 Nov 20. Review.

PMID:
25442121

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