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Items: 1 to 20 of 182

1.

Antisense oligonucleotide treatment ameliorates alpha-1 antitrypsin-related liver disease in mice.

Guo S, Booten SL, Aghajan M, Hung G, Zhao C, Blomenkamp K, Gattis D, Watt A, Freier SM, Teckman JH, McCaleb ML, Monia BP.

J Clin Invest. 2014 Jan;124(1):251-61. doi: 10.1172/JCI67968. Epub 2013 Dec 20.

2.

Knockdown of Z Mutant Alpha-1 Antitrypsin In Vivo Using Modified DNA Antisense Oligonucleotides.

Aghajan M, Guo S, Monia BP.

Methods Mol Biol. 2017;1639:127-138. doi: 10.1007/978-1-4939-7163-3_12.

PMID:
28752452
3.

Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency.

Borel F, Tang Q, Gernoux G, Greer C, Wang Z, Barzel A, Kay MA, Shultz LD, Greiner DL, Flotte TR, Brehm MA, Mueller C.

Mol Ther. 2017 Nov 1;25(11):2477-2489. doi: 10.1016/j.ymthe.2017.09.020. Epub 2017 Sep 25.

4.

Using antisense technology to develop a novel therapy for α-1 antitrypsin deficient (AATD) liver disease and to model AATD lung disease.

Guo S, Booten SL, Watt A, Alvarado L, Freier SM, Teckman JH, McCaleb ML, Monia BP.

Rare Dis. 2014 Mar 12;2:e28511. doi: 10.4161/rdis.28511. eCollection 2014.

5.

Amelioration of Alpha-1 Antitrypsin Deficiency Diseases with Genome Editing in Transgenic Mice.

Shen S, Sanchez ME, Blomenkamp K, Corcoran EM, Marco E, Yudkoff CJ, Jiang H, Teckman JH, Bumcrot D, Albright CF.

Hum Gene Ther. 2018 Aug;29(8):861-873. doi: 10.1089/hum.2017.227. Epub 2018 Jun 22.

PMID:
29641323
6.

Therapeutic Genome Editing With CRISPR/Cas9 in a Humanized Mouse Model Ameliorates α1-antitrypsin Deficiency Phenotype.

Bjursell M, Porritt MJ, Ericson E, Taheri-Ghahfarokhi A, Clausen M, Magnusson L, Admyre T, Nitsch R, Mayr L, Aasehaug L, Seeliger F, Maresca M, Bohlooly-Y M, Wiseman J.

EBioMedicine. 2018 Mar;29:104-111. doi: 10.1016/j.ebiom.2018.02.015. Epub 2018 Feb 19.

7.

Bone marrow stem cell therapy partially ameliorates pathological consequences in livers of mice expressing mutant human α1-antitrypsin.

Baligar P, Kochat V, Arindkar SK, Equbal Z, Mukherjee S, Patel S, Nagarajan P, Mohanty S, Teckman JH, Mukhopadhyay A.

Hepatology. 2017 Apr;65(4):1319-1335. doi: 10.1002/hep.29027. Epub 2017 Feb 25.

PMID:
28056498
8.

Hepatic-targeted RNA interference provides robust and persistent knockdown of alpha-1 antitrypsin levels in ZZ patients.

Turner AM, Stolk J, Bals R, Lickliter JD, Hamilton J, Christianson DR, Given BD, Burdon JG, Loomba R, Stoller JK, Teckman JH.

J Hepatol. 2018 Aug;69(2):378-384. doi: 10.1016/j.jhep.2018.03.012. Epub 2018 Mar 21.

PMID:
29572094
9.

Alpha-1-antitrypsin deficiency: from genoma to liver disease. PiZ mouse as model for the development of liver pathology in human.

Giovannoni I, Callea F, Stefanelli M, Mariani R, Santorelli FM, Francalanci P.

Liver Int. 2015 Jan;35(1):198-206. doi: 10.1111/liv.12504. Epub 2014 Mar 11.

PMID:
24529185
10.

In vivo post-transcriptional gene silencing of alpha-1 antitrypsin by adeno-associated virus vectors expressing siRNA.

Cruz PE, Mueller C, Cossette TL, Golant A, Tang Q, Beattie SG, Brantly M, Campbell-Thompson M, Blomenkamp KS, Teckman JH, Flotte TR.

Lab Invest. 2007 Sep;87(9):893-902. Epub 2007 Jun 25.

11.

Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector.

Conlon TJ, Cossette T, Erger K, Choi YK, Clarke T, Scott-Jorgensen M, Song S, Campbell-Thompson M, Crawford J, Flotte TR.

Mol Ther. 2005 Nov;12(5):867-75. Epub 2005 Aug 8.

12.

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
13.

Hereditary alpha-1-antitrypsin deficiency and its clinical consequences.

Fregonese L, Stolk J.

Orphanet J Rare Dis. 2008 Jun 19;3:16. doi: 10.1186/1750-1172-3-16. Review.

14.

Heteropolymerization of α-1-antitrypsin mutants in cell models mimicking heterozygosity.

Laffranchi M, Berardelli R, Ronzoni R, Lomas DA, Fra A.

Hum Mol Genet. 2018 May 15;27(10):1785-1793. doi: 10.1093/hmg/ddy090.

PMID:
29538751
15.

Autophagy induced by exogenous bile acids is therapeutic in a model of α-1-AT deficiency liver disease.

Tang Y, Fickert P, Trauner M, Marcus N, Blomenkamp K, Teckman J.

Am J Physiol Gastrointest Liver Physiol. 2016 Jul 1;311(1):G156-65. doi: 10.1152/ajpgi.00143.2015. Epub 2016 Apr 21.

16.

Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency.

Pastore N, Blomenkamp K, Annunziata F, Piccolo P, Mithbaokar P, Maria Sepe R, Vetrini F, Palmer D, Ng P, Polishchuk E, Iacobacci S, Polishchuk R, Teckman J, Ballabio A, Brunetti-Pierri N.

EMBO Mol Med. 2013 Mar;5(3):397-412. doi: 10.1002/emmm.201202046. Epub 2013 Feb 4.

17.

Liver Fibrosis and Metabolic Alterations in Adults With alpha-1-antitrypsin Deficiency Caused by the Pi*ZZ Mutation.

Hamesch K, Mandorfer M, Pereira VM, Moeller LS, Pons M, Dolman GE, Reichert MC, Schneider CV, Woditsch V, Voss J, Lindhauer C, Fromme M, Spivak I, Guldiken N, Zhou B, Arslanow A, Schaefer B, Zoller H, Aigner E, Reiberger T, Wetzel M, Siegmund B, Simões C, Gaspar R, Maia L, Costa D, Bento-Miranda M, van Helden J, Yagmur E, Bzdok D, Stolk J, Gleiber W, Knipel V, Windisch W, Mahadeva R, Bals R, Koczulla R, Barrecheguren M, Miravitlles M, Janciauskiene S, Stickel F, Lammert F, Liberal R, Genesca J, Griffiths WJ, Trauner M, Krag A, Trautwein C, Strnad P; European Alpha1-Liver Study Group.

Gastroenterology. 2019 Sep;157(3):705-719.e18. doi: 10.1053/j.gastro.2019.05.013. Epub 2019 May 20.

PMID:
31121167
18.

Hepatic progenitor cell proliferation and liver injury in α-1-antitrypsin deficiency.

Brunt EM, Blomenkamp K, Ahmed M, Ali F, Marcus N, Teckman J.

J Pediatr Gastroenterol Nutr. 2010 Nov;51(5):626-30. doi: 10.1097/MPG.0b013e3181e7ff55.

19.

Emerging Concepts and Human Trials in Alpha-1-Antitrypsin Deficiency Liver Disease.

Teckman JH.

Semin Liver Dis. 2017 May;37(2):152-158. doi: 10.1055/s-0037-1602586. Epub 2017 May 31. No abstract available.

PMID:
28564723
20.

Combination therapy utilizing shRNA knockdown and an optimized resistant transgene for rescue of diseases caused by misfolded proteins.

Li C, Xiao P, Gray SJ, Weinberg MS, Samulski RJ.

Proc Natl Acad Sci U S A. 2011 Aug 23;108(34):14258-63. doi: 10.1073/pnas.1109522108. Epub 2011 Aug 15.

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