메뉴 건너뛰기




Volumn 27, Issue 6, 2013, Pages 547-558

Alpha-1 antitrypsin deficiency: New developments in augmentation and other therapies

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA 1 ANTITRYPSIN; CARBAMAZEPINE; KAMADA API; LITHIUM; POLYMER; RAPAMYCIN; UNCLASSIFIED DRUG; VALPROIC ACID;

EID: 84891883736     PISSN: 11738804     EISSN: 1179190X     Source Type: Journal    
DOI: 10.1007/s40259-013-0042-5     Document Type: Article
Times cited : (10)

References (114)
  • 1
    • 84907041622 scopus 로고
    • The electrophoretic alpha 1 globulin pattern of serum in alpha 1 antitrypsin deficiency
    • 1:CAS:528:DyaF3sXksFOls7g%3D
    • Laurell CB, Eriksson S. The electrophoretic alpha 1 globulin pattern of serum in alpha 1 antitrypsin deficiency. Scand J Clin Lab Invest. 1963;15:132-40.
    • (1963) Scand J Clin Lab Invest , vol.15 , pp. 132-140
    • Laurell, C.B.1    Eriksson, S.2
  • 2
    • 0002203166 scopus 로고
    • Studies in alpha 1 antitrypsin deficiency
    • Eriksson S. Studies in alpha 1 antitrypsin deficiency. Acta Med Scand. 1965;177(Suppl):432.
    • (1965) Acta Med Scand , vol.177 , Issue.SUPPL.. , pp. 432
    • Eriksson, S.1
  • 3
    • 0019978932 scopus 로고
    • 1-antitrypsin
    • DOI 10.1038/298329a0
    • Carrell RW, Jeppsson JO, Laurell CB, Brennan SO, Owen MC, Vaughan L, et al. Structure and variation of human alpha 1-antitrypsin. Nature. 1982;298(5872):329-34. (Pubitemid 12044284)
    • (1982) Nature , vol.298 , Issue.5872 , pp. 329-334
    • Carrell, R.W.1    Jeppsson, J.O.2    Laurell, C.B.3
  • 4
    • 0019369133 scopus 로고
    • Emphysema induced in vitro and in vivo in dogs by a purified elastase from homologous leukocytes
    • Sloan B, Abrams WR, Meranze DR, Kimbel P, Weinbaum G. Emphysema induced in vitro and in vivo in dogs by a purified elastase from homologous leukocytes. Am Rev Respir Dis. 1981;124(3):295-301. (Pubitemid 11003995)
    • (1981) American Review of Respiratory Disease , vol.124 , Issue.3 , pp. 295-301
    • Sloan, B.1    Abrams, W.R.2    Meranze, D.R.3
  • 5
    • 0017669252 scopus 로고
    • The induction of pulmonary emphysema with human leukocyte elastase
    • Senior RM, Tegner H, Kuhn C, Ohlsson K, Starcher BC, Pierce JA. The induction of pulmonary emphysema with human leukocyte elastase. Am Rev Respir Dis. 1977;116(3):469-75. (Pubitemid 8181304)
    • (1977) American Review of Respiratory Disease , vol.116 , Issue.3 , pp. 469-475
    • Senior, R.M.1    Tegner, H.2    Kuhn, C.3
  • 6
    • 0021337902 scopus 로고
    • Emphysema and bronchial secretory cell metaplasia induced in hamsters by human neutrophil products
    • Snider GL, Lucey EC, Christensen TG, Stone PJ, Calore JD, Catanese A, et al. Emphysema and bronchial secretory cell metaplasia induced in hamsters by human neutrophil products. Am Rev Respir Dis. 1984;129(1):155-60. (Pubitemid 14176520)
    • (1984) American Review of Respiratory Disease , vol.129 , Issue.1 , pp. 155-160
    • Snider, G.L.1    Lucey, E.C.2    Christensen, T.G.3
  • 7
    • 0032717991 scopus 로고    scopus 로고
    • Neutrophils and protease/antiprotease imbalance
    • 10556170 1:STN:280:DC%2BD3c%2FksFSiuw%3D%3D 10.1164/ajrccm.160. supplement-1.13
    • Stockley RA. Neutrophils and protease/antiprotease imbalance. Am J Respir Crit Care Med. 1999;160(5 Pt 2):S49-52.
    • (1999) Am J Respir Crit Care Med , vol.160 , Issue.5 PART 2
    • Stockley, R.A.1
  • 8
    • 0026755363 scopus 로고
    • The mechanism of Z alpha 1-antitrypsin accumulation in the liver
    • 1608473 1:CAS:528:DyaK38Xks1Oqu7w%3D 10.1038/357605a0
    • Lomas DA, Evans DL, Finch JT, Carrell RW. The mechanism of Z alpha 1-antitrypsin accumulation in the liver. Nature. 1992;357(6379):605-7.
    • (1992) Nature , vol.357 , Issue.6379 , pp. 605-607
    • Lomas, D.A.1    Evans, D.L.2    Finch, J.T.3    Carrell, R.W.4
  • 9
    • 34547647784 scopus 로고    scopus 로고
    • Alpha one antitrypsin deficiency: From gene to treatment
    • DOI 10.1159/000105536
    • Wood AM, Stockley RA. Alpha one antitrypsin deficiency: from gene to treatment. Respiration. 2007;74(5):481-92. (Pubitemid 47206875)
    • (2007) Respiration , vol.74 , Issue.5 , pp. 481-492
    • Wood, A.M.1    Stockley, R.A.2
  • 10
    • 77950179576 scopus 로고    scopus 로고
    • Genetic variants of alpha1-antitrypsin
    • 19751191 1:CAS:528:DC%2BC3cXkt1eltr8%3D 10.2174/138920310790848368
    • Salahuddin P. Genetic variants of alpha1-antitrypsin. Curr Protein Pept Sci. 2010;11(2):101-17.
    • (2010) Curr Protein Pept Sci , vol.11 , Issue.2 , pp. 101-117
    • Salahuddin, P.1
  • 12
    • 0017099344 scopus 로고
    • Liver disease in alpha1-antitrypsin deficiency detected by screening of 200,000 infants
    • 1083485 1:STN:280:DyaE287nsVeltQ%3D%3D 10.1056/NEJM197606102942404
    • Sveger T. Liver disease in alpha1-antitrypsin deficiency detected by screening of 200,000 infants. N Engl J Med. 1976;294(24):1316-21.
    • (1976) N Engl J Med , vol.294 , Issue.24 , pp. 1316-1321
    • Sveger, T.1
  • 15
    • 67651204310 scopus 로고    scopus 로고
    • Oxidized {alpha}1-antitrypsin stimulates the release of monocyte chemotactic protein-1 from lung epithelial cells: Potential role in emphysema
    • 19525388 1:CAS:528:DC%2BD1MXhtVahtrrP 10.1152/ajplung.90373.2008
    • Li Z, Alam S, Wang J, Sandstrom CS, Janciauskiene S, Mahadeva R. Oxidized {alpha}1-antitrypsin stimulates the release of monocyte chemotactic protein-1 from lung epithelial cells: potential role in emphysema. Am J Physiol Lung Cell Mol Physiol. 2009;297(2):L388-400.
    • (2009) Am J Physiol Lung Cell Mol Physiol , vol.297 , Issue.2
    • Li, Z.1    Alam, S.2    Wang, J.3    Sandstrom, C.S.4    Janciauskiene, S.5    Mahadeva, R.6
  • 17
    • 80052478226 scopus 로고    scopus 로고
    • ANCA-associated vasculitis is linked to carriage of the Z allele of alpha(1) antitrypsin and its polymers
    • 21821620 1:CAS:528:DC%2BC3MXhtl2rsr3N 10.1136/ard.2011.153569
    • Morris H, Morgan MD, Wood AM, Smith SW, Ekeowa UI, Herrmann K, et al. ANCA-associated vasculitis is linked to carriage of the Z allele of alpha(1) antitrypsin and its polymers. Ann Rheum Dis. 2011;70(10):1851-6.
    • (2011) Ann Rheum Dis , vol.70 , Issue.10 , pp. 1851-1856
    • Morris, H.1    Morgan, M.D.2    Wood, A.M.3    Smith, S.W.4    Ekeowa, U.I.5    Herrmann, K.6
  • 18
    • 2442621622 scopus 로고    scopus 로고
    • 1-Antitrypsin deficiency·3: Clinical manifestations and natural history
    • DOI 10.1136/thx.2003.006510
    • Needham M, Stockley RA. Alpha 1-antitrypsin deficiency. 3: Clinical manifestations and natural history. Thorax. 2004;59(5):441-5. (Pubitemid 38624753)
    • (2004) Thorax , vol.59 , Issue.5 , pp. 441-445
    • Needham, M.1    Stockley, R.A.2
  • 19
    • 77955606635 scopus 로고    scopus 로고
    • Outdoor air pollution is associated with rapid decline of lung function in alpha-1-antitrypsin deficiency
    • 20519748 1:CAS:528:DC%2BC3cXhtV2rtbnK 10.1136/oem.2009.047589
    • Wood AM, Harrison RM, Semple S, Ayres JG, Stockley RA. Outdoor air pollution is associated with rapid decline of lung function in alpha-1-antitrypsin deficiency. Occup Environ Med. 2010;67(8):556-61.
    • (2010) Occup Environ Med , vol.67 , Issue.8 , pp. 556-561
    • Wood, A.M.1    Harrison, R.M.2    Semple, S.3    Ayres, J.G.4    Stockley, R.A.5
  • 20
    • 52649167915 scopus 로고    scopus 로고
    • The TNFalpha gene relates to clinical phenotype in alpha-1-antitrypsin deficiency
    • 18620570 10.1186/1465-9921-9-52 1:CAS:528:DC%2BD1cXhtFWgt7bK
    • Wood AM, Simmonds MJ, Bayley DL, Newby PR, Gough SC, Stockley RA. The TNFalpha gene relates to clinical phenotype in alpha-1-antitrypsin deficiency. Respir Res. 2008;9:52.
    • (2008) Respir Res , vol.9 , pp. 52
    • Wood, A.M.1    Simmonds, M.J.2    Bayley, D.L.3    Newby, P.R.4    Gough, S.C.5    Stockley, R.A.6
  • 21
    • 79951958144 scopus 로고    scopus 로고
    • Vitamin D-binding protein contributes to COPD by activation of alveolar macrophages
    • 21228423 1:STN:280:DC%2BC3M3gtF2rsA%3D%3D 10.1136/thx.2010.140921
    • Wood AM, Bassford C, Webster D, Newby P, Rajesh P, Stockley RA, et al. Vitamin D-binding protein contributes to COPD by activation of alveolar macrophages. Thorax. 2011;66(3):205-10.
    • (2011) Thorax , vol.66 , Issue.3 , pp. 205-210
    • Wood, A.M.1    Bassford, C.2    Webster, D.3    Newby, P.4    Rajesh, P.5    Stockley, R.A.6
  • 22
    • 84857229152 scopus 로고    scopus 로고
    • Association of IREB2 and CHRNA3 polymorphisms with airflow obstruction in severe alpha-1 antitrypsin deficiency
    • 22356581 1:CAS:528:DC%2BC38XlsVanu78%3D 10.1186/1465-9921-13-16
    • Kim WJ, Wood AM, Barker AF, Brantly ML, Campbell EJ, Eden E, et al. Association of IREB2 and CHRNA3 polymorphisms with airflow obstruction in severe alpha-1 antitrypsin deficiency. Respir Res. 2012;13:16.
    • (2012) Respir Res , vol.13 , pp. 16
    • Kim, W.J.1    Wood, A.M.2    Barker, A.F.3    Brantly, M.L.4    Campbell, E.J.5    Eden, E.6
  • 23
    • 27144471656 scopus 로고    scopus 로고
    • 1-antitrypsin deficiency: A continuing problem
    • DOI 10.1378/chest.128.4.1989
    • Stoller JK, Sandhaus RA, Turino G, Dickson R, Rodgers K, Strange C. Delay in diagnosis of alpha1-antitrypsin deficiency: a continuing problem. Chest. 2005;128(4):1989-94. (Pubitemid 41507532)
    • (2005) Chest , vol.128 , Issue.4 , pp. 1989-1994
    • Stoller, J.K.1    Sandhaus, R.A.2    Turino, G.3    Dickson, R.4    Rodgers, K.5    Strange, C.6
  • 24
    • 84874708546 scopus 로고    scopus 로고
    • Age related development of respiratory abnormalities in non-index alpha-1 antitrypsin deficient studies
    • 23276486 10.1016/j.rmed.2012.12.003
    • Holme J, Stockley JA, Stockley RA. Age related development of respiratory abnormalities in non-index alpha-1 antitrypsin deficient studies. Respir Med. 2013;107(3):387-93.
    • (2013) Respir Med , vol.107 , Issue.3 , pp. 387-393
    • Holme, J.1    Stockley, J.A.2    Stockley, R.A.3
  • 26
    • 0019787989 scopus 로고
    • Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjects
    • Gadek JE, Klein HG, Holland PV, Crystal RG. Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjects. J Clin Investig. 1981;68(5):1158-65. (Pubitemid 12235015)
    • (1981) Journal of Clinical Investigation , vol.68 , Issue.5 , pp. 1158-1165
    • Gadek, J.E.1    Klein, H.G.2    Holland, P.V.3    Crystal, R.G.4
  • 27
    • 0023148995 scopus 로고
    • 1-antitrypsin deficiency associated with emphysema
    • Wewers MD, Casolaro MA, Sellers SE, Swayze SC, McPhaul KM, Wittes JT, et al. Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema. N Engl J Med. 1987;316(17):1055-62. (Pubitemid 17047294)
    • (1987) New England Journal of Medicine , vol.316 , Issue.17 , pp. 1055-1062
    • Wewers, M.D.1    Casolaro, M.A.2    Sellers, S.E.3
  • 28
    • 0141706635 scopus 로고    scopus 로고
    • American Thoracic Society/European Respiratory Society statement: Standards for the diagnosis and management of individuals with alpha-1 antitrypsin deficiency
    • American Thoracic Society/European Respiratory Society
    • American Thoracic Society/European Respiratory Society. American Thoracic Society/European Respiratory Society statement: standards for the diagnosis and management of individuals with alpha-1 antitrypsin deficiency. Am J Respir Crit Care Med. 2003;168(7):818-900.
    • (2003) Am J Respir Crit Care Med , vol.168 , Issue.7 , pp. 818-900
  • 29
    • 0031242842 scopus 로고    scopus 로고
    • 1- antitrypsin deficiency?
    • DOI 10.1183/09031936.97.10102260
    • Seersholm N, Wencker M, Banik N, Viskum K, Dirksen A, Kok-Jensen A, et al. Does alpha1-antitrypsin augmentation therapy slow the annual decline in FEV1 in patients with severe hereditary alpha1-antitrypsin deficiency? Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL) alpha1-AT study group. Eur Respir J. 1997;10(10):2260-3. (Pubitemid 27479546)
    • (1997) European Respiratory Journal , vol.10 , Issue.10 , pp. 2260-2263
    • Seersholm, N.1    Wencker, M.2    Banik, N.3    Viskum, K.4    Dirksen, A.5    Kok-Jensen, A.6    Konietzko, N.7
  • 30
    • 0031884479 scopus 로고    scopus 로고
    • 1-antitrypsin
    • DOI 10.1183/09031936.98.11020428
    • Wencker M, Banik N, Buhl R, Seidel R, Konietzko N. Long-term treatment of alpha1-antitrypsin deficiency-related pulmonary emphysema with human alpha1-antitrypsin. Wissenschaftliche Arbeitsgemeinschaft zur Therapie von Lungenerkrankungen (WATL)-alpha1-AT-study group. Eur Respir J. 1998;11(2):428-33. (Pubitemid 28108360)
    • (1998) European Respiratory Journal , vol.11 , Issue.2 , pp. 428-433
    • Wencker, M.1    Banik, N.2    Buhl, R.3    Seidel, R.4    Konietzko, N.5
  • 31
    • 84859354170 scopus 로고    scopus 로고
    • Reduction of severe exacerbations and hospitalization-derived costs in alpha-1-antitrypsin-deficient patients treated with alpha-1-antitrypsin augmentation therapy
    • 22354900 1:CAS:528:DC%2BC38Xot1egtb0%3D 10.1177/1753465812438387
    • Barros-Tizon JC, Torres ML, Blanco I, Martinez MT. Reduction of severe exacerbations and hospitalization-derived costs in alpha-1-antitrypsin-deficient patients treated with alpha-1-antitrypsin augmentation therapy. Ther Adv Respir Dis. 2012;6(2):67-78.
    • (2012) Ther Adv Respir Dis , vol.6 , Issue.2 , pp. 67-78
    • Barros-Tizon, J.C.1    Torres, M.L.2    Blanco, I.3    Martinez, M.T.4
  • 32
    • 0032110436 scopus 로고    scopus 로고
    • Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin
    • The Alpha-1-Antitrypsin Deficiency Registry Study Group
    • Survival and FEV1 decline in individuals with severe deficiency of alpha1-antitrypsin. The Alpha-1-Antitrypsin Deficiency Registry Study Group. Am J Respir Crit Care Med. 1998;158(1):49-59.
    • (1998) Am J Respir Crit Care Med. , vol.158 , Issue.1 , pp. 49-59
  • 33
    • 0032724157 scopus 로고    scopus 로고
    • A randomized clinical trial of alpha(1)-antitrypsin augmentation therapy
    • 10556107 1:STN:280:DC%2BD3c%2FislKltg%3D%3D 10.1164/ajrccm.160.5.9901055
    • Dirksen A, Dijkman JH, Madsen F, Stoel B, Hutchison DC, Ulrik CS, et al. A randomized clinical trial of alpha(1)-antitrypsin augmentation therapy. Am J Respir Crit Care Med. 1999;160(5 Pt 1):1468-72.
    • (1999) Am J Respir Crit Care Med , vol.160 , Issue.5 PART 1 , pp. 1468-1472
    • Dirksen, A.1    Dijkman, J.H.2    Madsen, F.3    Stoel, B.4    Hutchison, D.C.5    Ulrik, C.S.6
  • 34
    • 67649407825 scopus 로고    scopus 로고
    • Exploring the role of CT densitometry: A randomised study of augmentation therapy in alpha1-antitrypsin deficiency
    • 19196813 1:CAS:528:DC%2BD1MXoslyht70%3D 10.1183/09031936.00159408
    • Dirksen A, Piitulainen E, Parr DG, Deng C, Wencker M, Shaker SB, et al. Exploring the role of CT densitometry: a randomised study of augmentation therapy in alpha1-antitrypsin deficiency. Eur Respir J. 2009;33(6):1345-53.
    • (2009) Eur Respir J , vol.33 , Issue.6 , pp. 1345-1353
    • Dirksen, A.1    Piitulainen, E.2    Parr, D.G.3    Deng, C.4    Wencker, M.5    Shaker, S.B.6
  • 35
    • 77955487511 scopus 로고    scopus 로고
    • Intravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease
    • Epub 2010/07/09
    • Gotzsche PC, Johansen HK. Intravenous alpha-1 antitrypsin augmentation therapy for treating patients with alpha-1 antitrypsin deficiency and lung disease. Cochrane Database Syst Rev. 2010;(7):CD007851. Epub 2010/07/09.
    • (2010) Cochrane Database Syst Rev. , Issue.7
    • Gotzsche, P.C.1    Johansen, H.K.2
  • 36
    • 78649455530 scopus 로고    scopus 로고
    • Exploring the optimum approach to the use of CT densitometry in a randomised placebo-controlled study of augmentation therapy in alpha 1-antitrypsin deficiency
    • 19678952 10.1186/1465-9921-10-75 1:CAS:528:DC%2BD1MXhtVemurfM
    • Parr DG, Dirksen A, Piitulainen E, Deng C, Wencker M, Stockley RA. Exploring the optimum approach to the use of CT densitometry in a randomised placebo-controlled study of augmentation therapy in alpha 1-antitrypsin deficiency. Respir Res. 2009;10:75.
    • (2009) Respir Res , vol.10 , pp. 75
    • Parr, D.G.1    Dirksen, A.2    Piitulainen, E.3    Deng, C.4    Wencker, M.5    Stockley, R.A.6
  • 37
    • 77957273246 scopus 로고    scopus 로고
    • Therapeutic efficacy of alpha-1 antitrypsin augmentation therapy on the loss of lung tissue: An integrated analysis of 2 randomised clinical trials using computed tomography densitometry
    • 20920370 10.1186/1465-9921-11-136 1:CAS:528:DC%2BC3cXht1KlsLfO
    • Stockley RA, Parr DG, Piitulainen E, Stolk J, Stoel BC, Dirksen A. Therapeutic efficacy of alpha-1 antitrypsin augmentation therapy on the loss of lung tissue: an integrated analysis of 2 randomised clinical trials using computed tomography densitometry. Respir Res. 2010;11:136.
    • (2010) Respir Res , vol.11 , pp. 136
    • Stockley, R.A.1    Parr, D.G.2    Piitulainen, E.3    Stolk, J.4    Stoel, B.C.5    Dirksen, A.6
  • 38
    • 70350450961 scopus 로고    scopus 로고
    • Augmentation therapy for alpha1 antitrypsin deficiency: A meta-analysis
    • 19811373 10.1080/15412550902905961
    • Chapman KR, Stockley RA, Dawkins C, Wilkes MM, Navickis RJ. Augmentation therapy for alpha1 antitrypsin deficiency: a meta-analysis. COPD. 2009;6(3):177-84.
    • (2009) COPD , vol.6 , Issue.3 , pp. 177-184
    • Chapman, K.R.1    Stockley, R.A.2    Dawkins, C.3    Wilkes, M.M.4    Navickis, R.J.5
  • 39
    • 0023784904 scopus 로고
    • Biochemical efficacy and safety of monthly augmentation therapy for alpha 1-antitrypsin deficiency
    • 3261353 1:STN:280:DyaL1czgtV2rtA%3D%3D 10.1001/jama.1988.03410090091037
    • Hubbard RC, Sellers S, Czerski D, Stephens L, Crystal RG. Biochemical efficacy and safety of monthly augmentation therapy for alpha 1-antitrypsin deficiency. JAMA. 1988;260(9):1259-64.
    • (1988) JAMA , vol.260 , Issue.9 , pp. 1259-1264
    • Hubbard, R.C.1    Sellers, S.2    Czerski, D.3    Stephens, L.4    Crystal, R.G.5
  • 41
    • 42949085601 scopus 로고    scopus 로고
    • 1-antitrypsin deficiency: From pharmacokinetic analysis to individualizing therapy
    • DOI 10.1345/aph.1K505
    • Zamora NP, Pla RV, Del Rio PG, Margaleff RJ, Frias FR, Ronsano JB. Intravenous human plasma-derived augmentation therapy in alpha 1-antitrypsin deficiency: from pharmacokinetic analysis to individualizing therapy. Ann Pharmacother. 2008;42(5):640-6. (Pubitemid 351619934)
    • (2008) Annals of Pharmacotherapy , vol.42 , Issue.5 , pp. 640-646
    • Zamora, N.P.1    Pla, R.V.2    Del Rio, P.G.3    Margaleff, R.J.4    Frias, F.R.5    Ronsano, J.B.M.6
  • 42
    • 77957201481 scopus 로고    scopus 로고
    • Pharmacokinetic comparability of Prolastin(R)-C to Prolastin(R) in alpha(1)-antitrypsin deficiency: A randomized study
    • 20920295 10.1186/1472-6904-10-13 1:CAS:528:DC%2BC3cXht1ygsbnI
    • Stocks JM, Brantly ML, Wang-Smith L, Campos MA, Chapman KR, Kueppers F, et al. Pharmacokinetic comparability of Prolastin(R)-C to Prolastin(R) in alpha(1)-antitrypsin deficiency: a randomized study. BMC Clin Pharmacol. 2010;10:13.
    • (2010) BMC Clin Pharmacol , vol.10 , pp. 13
    • Stocks, J.M.1    Brantly, M.L.2    Wang-Smith, L.3    Campos, M.A.4    Chapman, K.R.5    Kueppers, F.6
  • 43
    • 33845227581 scopus 로고    scopus 로고
    • Alpha-1-antitrypsin deficiency: Optimal therapeutic regimen based on population pharmacokinetics
    • DOI 10.1136/thx.2005.057943
    • Soy D, de la Roza C, Lara B, Esquinas C, Torres A, Miravitlles M. Alpha-1-antitrypsin deficiency: optimal therapeutic regimen based on population pharmacokinetics. Thorax. 2006;61(12):1059-64. (Pubitemid 44852024)
    • (2006) Thorax , vol.61 , Issue.12 , pp. 1059-1064
    • Soy, D.1    De La Roza, C.2    Lara, B.3    Esquinas, C.4    Torres, A.5    Miravitlles, M.6
  • 44
    • 84861316814 scopus 로고    scopus 로고
    • Alpha-1 antitrypsin deficiency targeted testing and augmentation therapy: A Canadian Thoracic Society clinical practice guideline
    • 22536580
    • Marciniuk DD, Hernandez P, Balter M, Bourbeau J, Chapman KR, Ford GT, et al. Alpha-1 antitrypsin deficiency targeted testing and augmentation therapy: a Canadian Thoracic Society clinical practice guideline. Can Respir J. 2012;19(2):109-16.
    • (2012) Can Respir J , vol.19 , Issue.2 , pp. 109-116
    • Marciniuk, D.D.1    Hernandez, P.2    Balter, M.3    Bourbeau, J.4    Chapman, K.R.5    Ford, G.T.6
  • 45
    • 69249228595 scopus 로고    scopus 로고
    • Mortality in alpha-1-antitrypsin deficiency in the United Kingdom
    • 19443188 10.1016/j.rmed.2009.04.004
    • Dawkins P, Wood A, Nightingale P, Stockley R. Mortality in alpha-1-antitrypsin deficiency in the United Kingdom. Respir Med. 2009;103(10):1540-7.
    • (2009) Respir Med , vol.103 , Issue.10 , pp. 1540-1547
    • Dawkins, P.1    Wood, A.2    Nightingale, P.3    Stockley, R.4
  • 46
    • 77954639932 scopus 로고    scopus 로고
    • Survival in severe alpha-1-antitrypsin deficiency (PiZZ)
    • 20420704 10.1186/1465-9921-11-44
    • Tanash HA, Nilsson PM, Nilsson JA, Piitulainen E. Survival in severe alpha-1-antitrypsin deficiency (PiZZ). Respir Res. 2010;11:44.
    • (2010) Respir Res , vol.11 , pp. 44
    • Tanash, H.A.1    Nilsson, P.M.2    Nilsson, J.A.3    Piitulainen, E.4
  • 47
    • 69249101657 scopus 로고    scopus 로고
    • Rate of progression of lung function impairment in alpha1-antitrypsin deficiency
    • 19164359 1:STN:280:DC%2BD1MzosVWjsA%3D%3D 10.1183/09031936.00061208
    • Dawkins PA, Dawkins CL, Wood AM, Nightingale PG, Stockley JA, Stockley RA. Rate of progression of lung function impairment in alpha1-antitrypsin deficiency. Eur Respir J. 2009;33(6):1338-44.
    • (2009) Eur Respir J , vol.33 , Issue.6 , pp. 1338-1344
    • Dawkins, P.A.1    Dawkins, C.L.2    Wood, A.M.3    Nightingale, P.G.4    Stockley, J.A.5    Stockley, R.A.6
  • 48
    • 79953858686 scopus 로고    scopus 로고
    • Efficacy of alpha1-antitrypsin augmentation therapy in conditions other than pulmonary emphysema
    • 21486454 10.1186/1750-1172-6-14
    • 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.
    • (2011) Orphanet J Rare Dis , vol.6 , pp. 14
    • Blanco, I.1    Lara, B.2    De Serres, F.3
  • 49
    • 0025360692 scopus 로고
    • 1-antitrypsin deficiency by the aerosol route
    • Hubbard RC, Crystal RG. Strategies for aerosol therapy of alpha 1-antitrypsin deficiency by the aerosol route. Lung. 1990;168(Suppl):565-78. (Pubitemid 20217698)
    • (1990) Lung , vol.168 , Issue.SUPPL.. , pp. 565-578
    • Hubbard, R.C.1    Crystal, R.G.2
  • 52
    • 69249120315 scopus 로고    scopus 로고
    • Lung deposition of inhaled alpha1-proteinase inhibitor in cystic fibrosis and alpha1-antitrypsin deficiency
    • 19251783 1:CAS:528:DC%2BD1MXhtVCht77J 10.1183/09031936.00118408
    • Brand P, Schulte M, Wencker M, Herpich CH, Klein G, Hanna K, et al. Lung deposition of inhaled alpha1-proteinase inhibitor in cystic fibrosis and alpha1-antitrypsin deficiency. Eur Respir J. 2009;34(2):354-60.
    • (2009) Eur Respir J , vol.34 , Issue.2 , pp. 354-360
    • Brand, P.1    Schulte, M.2    Wencker, M.3    Herpich, C.H.4    Klein, G.5    Hanna, K.6
  • 53
    • 21244492343 scopus 로고    scopus 로고
    • A pilot study comparing the purity, functionality and isoform composition of alpha-1-proteinase inhibitor (human) products
    • DOI 10.1185/030079905X46395, 3011
    • Cowden DI, Fisher GE, Weeks RL. A pilot study comparing the purity, functionality and isoform composition of alpha-1-proteinase inhibitor (human) products. Curr Med Res Opin. 2005;21(6):877-83. (Pubitemid 40896236)
    • (2005) Current Medical Research and Opinion , vol.21 , Issue.6 , pp. 877-883
    • Cowden, D.I.1    Fisher, G.E.2    Weeks, R.L.3
  • 54
    • 33745065087 scopus 로고    scopus 로고
    • Recombinant human alpha-1 proteinase inhibitor: Towards therapeutic use
    • DOI 10.1007/s00726-005-0324-4
    • Karnaukhova E, Ophir Y, Golding B. Recombinant human alpha-1 proteinase inhibitor: towards therapeutic use. Amino Acids. 2006;30(4):317-32. (Pubitemid 43886895)
    • (2006) Amino Acids , vol.30 , Issue.4 , pp. 317-332
    • Karnaukhova, E.1    Ophir, Y.2    Golding, B.3
  • 56
    • 0002175173 scopus 로고    scopus 로고
    • Cell surface carbohydrates: Cell type-specific expression
    • M. Fukada O. Hinghaul (eds) Oxford University Press New York
    • Fukada M. Cell surface carbohydrates: cell type-specific expression. In: Fukada M, Hinghaul O, editors. Molecular and cellular glycobiology. New York: Oxford University Press; 2000. p. 12-61.
    • (2000) Molecular and Cellular Glycobiology , pp. 12-61
    • Fukada, M.1
  • 57
    • 0036274761 scopus 로고    scopus 로고
    • 1-proteinase inhibitor and improved anti-elastase activity of the inhibitor with glutamate at position 351
    • Sandoval C, Curtis H, Congote LF. Enhanced proliferative effects of a baculovirus-produced fusion protein of insulin-like growth factor and alpha(1)-proteinase inhibitor and improved anti-elastase activity of the inhibitor with glutamate at position 351. Protein Eng. 2002;15(5):413-8. (Pubitemid 34601733)
    • (2002) Protein Engineering , vol.15 , Issue.5 , pp. 413-418
    • Sandoval, C.1    Curtis, H.2    Congote, L.F.3
  • 58
    • 0027521818 scopus 로고
    • Transgenic livestock as bioreactors: Stable expression of human alpha-1-antitrypsin by a flock of sheep
    • 1:CAS:528:DyaK2cXnsFGitQ%3D%3D 10.1038/nbt1193-1263
    • Carver AS, Dalrymple MA, Wright G, Cottom DS, Reeves DB, Gibson YH, et al. Transgenic livestock as bioreactors: stable expression of human alpha-1-antitrypsin by a flock of sheep. Biotechnology (N Y). 1993;11(11):1263-70.
    • (1993) Biotechnology (N Y) , vol.11 , Issue.11 , pp. 1263-1270
    • Carver, A.S.1    Dalrymple, M.A.2    Wright, G.3    Cottom, D.S.4    Reeves, D.B.5    Gibson, Y.H.6
  • 59
    • 14744276106 scopus 로고
    • High level expression of active human alpha-1-antitrypsin in the milk of transgenic sheep
    • 1:CAS:528:DyaK38Xht1aitLk%3D 10.1038/nbt0991-830
    • Wright G, Carver A, Cottom D, Reeves D, Scott A, Simons P, et al. High level expression of active human alpha-1-antitrypsin in the milk of transgenic sheep. Biotechnology (N Y). 1991;9(9):830-4.
    • (1991) Biotechnology (N Y) , vol.9 , Issue.9 , pp. 830-834
    • Wright, G.1    Carver, A.2    Cottom, D.3    Reeves, D.4    Scott, A.5    Simons, P.6
  • 62
    • 0026725682 scopus 로고
    • A 17.6 kbp region located upstream of the rabbit WAP gene directs high level expression of a functional human protein variant in transgenic mouse milk
    • 1299629 1:CAS:528:DyaK38XltVGgtLs%3D 10.1016/0014-5793(92)80683-8
    • Bischoff R, Degryse E, Perraud F, Dalemans W, Ali-Hadji D, Thepot D, et al. A 17.6 kbp region located upstream of the rabbit WAP gene directs high level expression of a functional human protein variant in transgenic mouse milk. FEBS Lett. 1992;305(3):265-8.
    • (1992) FEBS Lett , vol.305 , Issue.3 , pp. 265-268
    • Bischoff, R.1    Degryse, E.2    Perraud, F.3    Dalemans, W.4    Ali-Hadji, D.5    Thepot, D.6
  • 64
    • 0025732278 scopus 로고
    • Expression of active recombinant human alpha 1-antitrypsin in transgenic rabbits
    • 1367260 1:CAS:528:DyaK3MXkt1els78%3D 10.1016/0168-1656(91)90247-S
    • Massoud M, Bischoff R, Dalemans W, Pointu H, Attal J, Schultz H, et al. Expression of active recombinant human alpha 1-antitrypsin in transgenic rabbits. J Biotechnol. 1991;18(3):193-203.
    • (1991) J Biotechnol , vol.18 , Issue.3 , pp. 193-203
    • Massoud, M.1    Bischoff, R.2    Dalemans, W.3    Pointu, H.4    Attal, J.5    Schultz, H.6
  • 65
    • 0030032878 scopus 로고    scopus 로고
    • Low levels of chimerism in rabbit fetuses produced from preimplantation embryos microinjected with fetal gonadal cells
    • DOI 10.1002/(SICI)1098-2795(199601) 43:1<38::AID-MRD5>3.0.CO;2-V
    • Moens A, Betteridge KJ, Brunet A, Renard JP. Low levels of chimerism in rabbit fetuses produced from preimplantation embryos microinjected with fetal gonadal cells. Mol Reprod Dev. 1996;43(1):38-46. (Pubitemid 26021945)
    • (1996) Molecular Reproduction and Development , vol.43 , Issue.1 , pp. 38-46
    • Moens, A.1    Betteridge, K.J.2    Brunet, A.3    Renard, J.-P.4
  • 66
  • 67
    • 67049137904 scopus 로고    scopus 로고
    • First US approval for a transgenic animal drug
    • 19352350 1:CAS:528:DC%2BD1MXktFaksLo%3D 10.1038/nbt0409-302
    • Kling J. First US approval for a transgenic animal drug. Nat Biotechnol. 2009;27(4):302-4.
    • (2009) Nat Biotechnol , vol.27 , Issue.4 , pp. 302-304
    • Kling, J.1
  • 68
    • 0017808477 scopus 로고
    • 1-anti-trypsin in rat circulation
    • DOI 10.1016/0020-711X(78)90020-4
    • Yu SD, Gan JC. Effects of progressive desialylation on the survival of human plasma alpha1-anti-trypsin in rat circulation. Int J Biochem. 1978;9(2):107-15. (Pubitemid 8301974)
    • (1978) International Journal of Biochemistry , vol.9 , Issue.2 , pp. 107-115
    • Yu, S.D.1    Gan, J.C.2
  • 69
    • 84868502136 scopus 로고    scopus 로고
    • Production and characterization of a novel human recombinant alpha-1-antitrypsin in PER.C6 cells
    • 23036927 1:CAS:528:DC%2BC38Xhslejtb3L 10.1016/j.jbiotec.2012.09.018
    • Ross D, Brown T, Harper R, Pamarthi M, Nixon J, Bromirski J, et al. Production and characterization of a novel human recombinant alpha-1-antitrypsin in PER.C6 cells. J Biotechnol. 2012;162(2-3):262-73.
    • (2012) J Biotechnol , vol.162 , Issue.2-3 , pp. 262-273
    • Ross, D.1    Brown, T.2    Harper, R.3    Pamarthi, M.4    Nixon, J.5    Bromirski, J.6
  • 70
    • 84875384860 scopus 로고    scopus 로고
    • Structural characterization of recombinant alpha-1-antitrypsin expressed in a human cell line
    • Epub 2013/03/07
    • Wang Z, Hilder TL, Drift KV, Sloan J, Wee K. Structural characterization of recombinant alpha-1-antitrypsin expressed in a human cell line. Anal Biochem. 2013. Epub 2013/03/07.
    • (2013) Anal Biochem
    • Wang, Z.1    Hilder, T.L.2    Drift, K.V.3    Sloan, J.4    Wee, K.5
  • 71
    • 0029048542 scopus 로고
    • The Z type variation of human alpha 1-antitrypsin causes a protein folding defect
    • 7664092 1:CAS:528:DyaK2MXlsFKltr8%3D 10.1038/nsb0595-363
    • Yu MH, Lee KN, Kim J. The Z type variation of human alpha 1-antitrypsin causes a protein folding defect. Nat Struct Biol. 1995;2(5):363-7.
    • (1995) Nat Struct Biol , vol.2 , Issue.5 , pp. 363-367
    • Yu, M.H.1    Lee, K.N.2    Kim, J.3
  • 72
    • 80051635385 scopus 로고    scopus 로고
    • Oxidation of Z alpha1-antitrypsin by cigarette smoke induces polymerization: A novel mechanism of early-onset emphysema
    • 20971880 1:CAS:528:DC%2BC3MXhtVSitrrL 10.1165/rcmb.2010-0328OC
    • Alam S, Li Z, Janciauskiene S, Mahadeva R. Oxidation of Z alpha1-antitrypsin by cigarette smoke induces polymerization: a novel mechanism of early-onset emphysema. Am J Respir Cell Mol Biol. 2011;45(2):261-9.
    • (2011) Am J Respir Cell Mol Biol , vol.45 , Issue.2 , pp. 261-269
    • Alam, S.1    Li, Z.2    Janciauskiene, S.3    Mahadeva, R.4
  • 73
    • 0034652248 scopus 로고    scopus 로고
    • Chemical chaperones mediate increased secretion of mutant α1- antitrypsin (α1-AT) Z: A potential pharmacological strategy for prevention of liver injury and emphysema in α1-AT deficiency
    • DOI 10.1073/pnas.97.4.1796
    • Burrows JA, Willis LK, Perlmutter DH. Chemical chaperones mediate increased secretion of mutant alpha 1-antitrypsin (alpha 1-AT) Z: a potential pharmacological strategy for prevention of liver injury and emphysema in alpha 1-AT deficiency. Proc Natl Acad Sci USA. 2000;97(4):1796-801. (Pubitemid 30118522)
    • (2000) Proceedings of the National Academy of Sciences of the United States of America , vol.97 , Issue.4 , pp. 1796-1801
    • Burrows, J.A.J.1    Willis, L.K.2    Perlmutter, D.H.3
  • 77
    • 0036510604 scopus 로고    scopus 로고
    • 1-antitrypsin-related cirrhosis
    • DOI 10.1074/jbc.C100722200
    • Mahadeva R, Dafforn TR, Carrell RW, Lomas DA. 6-mer peptide selectively anneals to a pathogenic serpin conformation and blocks polymerization. Implications for the prevention of Z alpha(1)-antitrypsin-related cirrhosis. J Biol Chem. 2002;277(9):6771-4. (Pubitemid 34953057)
    • (2002) Journal of Biological Chemistry , vol.277 , Issue.9 , pp. 6771-6774
    • Mahadeva, R.1    Dafforn, T.R.2    Carrell, R.W.3    Lomas, D.A.4
  • 80
    • 84862660301 scopus 로고    scopus 로고
    • Preventing and reversing the cellular consequences of Z alpha-1 antitrypsin accumulation by targeting s4A
    • 22425623 1:CAS:528:DC%2BC38XovV2guro%3D 10.1016/j.jhep.2012.02.025
    • Alam S, Wang J, Janciauskiene S, Mahadeva R. Preventing and reversing the cellular consequences of Z alpha-1 antitrypsin accumulation by targeting s4A. J Hepatol. 2012;57(1):116-24.
    • (2012) J Hepatol , vol.57 , Issue.1 , pp. 116-124
    • Alam, S.1    Wang, J.2    Janciauskiene, S.3    Mahadeva, R.4
  • 81
    • 84868611206 scopus 로고    scopus 로고
    • The emerging importance of autophagy in pulmonary diseases
    • 23131937 1:CAS:528:DC%2BC3sXnslCl 10.1378/chest.12-0809
    • Mizumura K, Cloonan SM, Haspel JA, Choi AM. The emerging importance of autophagy in pulmonary diseases. Chest. 2012;142(5):1289-99.
    • (2012) Chest , vol.142 , Issue.5 , pp. 1289-1299
    • Mizumura, K.1    Cloonan, S.M.2    Haspel, J.A.3    Choi, A.M.4
  • 82
    • 77954597127 scopus 로고    scopus 로고
    • An autophagy-enhancing drug promotes degradation of mutant alpha1-antitrypsin Z and reduces hepatic fibrosis
    • 20522742 1:CAS:528:DC%2BC3cXosVWgu70%3D 10.1126/science.1190354
    • Hidvegi T, Ewing M, Hale P, Dippold C, Beckett C, Kemp C, et al. An autophagy-enhancing drug promotes degradation of mutant alpha1-antitrypsin Z and reduces hepatic fibrosis. Science. 2010;329(5988):229-32.
    • (2010) Science , vol.329 , Issue.5988 , pp. 229-232
    • Hidvegi, T.1    Ewing, M.2    Hale, P.3    Dippold, C.4    Beckett, C.5    Kemp, C.6
  • 83
    • 0026586140 scopus 로고
    • Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes
    • 1729724 1:CAS:528:DyaK38Xns1egtA%3D%3D 10.1073/pnas.89.1.89
    • Kay MA, Baley P, Rothenberg S, Leland F, Fleming L, Ponder KP, et al. Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci USA. 1992;89(1):89-93.
    • (1992) Proc Natl Acad Sci USA , vol.89 , Issue.1 , pp. 89-93
    • Kay, M.A.1    Baley, P.2    Rothenberg, S.3    Leland, F.4    Fleming, L.5    Ponder, K.P.6
  • 84
    • 0026906987 scopus 로고
    • Adenovirus-mediated in vivo gene transfer and expression in normal rat liver
    • 1302034 1:CAS:528:DyaK38Xls1Gjs78%3D 10.1038/ng0892-372
    • Jaffe HA, Danel C, Longenecker G, Metzger M, Setoguchi Y, Rosenfeld MA, et al. Adenovirus-mediated in vivo gene transfer and expression in normal rat liver. Nat Genet. 1992;1(5):372-8.
    • (1992) Nat Genet , vol.1 , Issue.5 , pp. 372-378
    • Jaffe, H.A.1    Danel, C.2    Longenecker, G.3    Metzger, M.4    Setoguchi, Y.5    Rosenfeld, M.A.6
  • 88
    • 0028899573 scopus 로고
    • Therapeutic serum concentrations of human alpha-1-antitrypsin after adenoviral-mediated gene transfer into mouse hepatocytes
    • 7875680 1:CAS:528:DyaK2MXltlentL0%3D
    • Kay MA, Graham F, Leland F, Woo SL. Therapeutic serum concentrations of human alpha-1-antitrypsin after adenoviral-mediated gene transfer into mouse hepatocytes. Hepatology. 1995;21(3):815-9.
    • (1995) Hepatology , vol.21 , Issue.3 , pp. 815-819
    • Kay, M.A.1    Graham, F.2    Leland, F.3    Woo, S.L.4
  • 89
    • 33745241652 scopus 로고    scopus 로고
    • Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector
    • 16518879 1:CAS:528:DC%2BD28XnsVWjuro%3D 10.1002/jgm.896
    • Lu Y, Choi YK, Campbell-Thompson M, Li C, Tang Q, Crawford JM, et al. Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector. J Gene Med. 2006;8(6):730-5.
    • (2006) J Gene Med , vol.8 , Issue.6 , pp. 730-735
    • Lu, Y.1    Choi, Y.K.2    Campbell-Thompson, M.3    Li, C.4    Tang, Q.5    Crawford, J.M.6
  • 90
    • 0034828903 scopus 로고    scopus 로고
    • Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors
    • DOI 10.1038/sj.gt.3301422
    • Song S, Embury J, Laipis PJ, Berns KI, Crawford JM, Flotte TR. Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors. Gene Ther. 2001;8(17):1299-306. (Pubitemid 32834298)
    • (2001) Gene Therapy , vol.8 , Issue.17 , pp. 1299-1306
    • Song, S.1    Embury, J.2    Laipis, P.J.3    Berns, K.I.4    Crawford, J.M.5    Flotte, T.R.6
  • 92
    • 0028172344 scopus 로고
    • Expression of human alpha 1-antitrypsin in mouse after in vivo gene transfer to hepatocytes by small liposomes
    • 7980573 1:CAS:528:DyaK2MXitV2gtr4%3D 10.1006/bbrc.1994.2565
    • Alino SF, Crespo J, Bobadilla M, Lejarreta M, Blaya C, Crespo A. Expression of human alpha 1-antitrypsin in mouse after in vivo gene transfer to hepatocytes by small liposomes. Biochem Biophys Res Commun. 1994;204(3):1023-30.
    • (1994) Biochem Biophys Res Commun , vol.204 , Issue.3 , pp. 1023-1030
    • Alino, S.F.1    Crespo, J.2    Bobadilla, M.3    Lejarreta, M.4    Blaya, C.5    Crespo, A.6
  • 93
    • 0028056371 scopus 로고
    • Aerosol and intravenous transfection of human alpha 1-antitrypsin gene to lungs of rabbits
    • 8292378 1:CAS:528:DyaK2cXlslWls7g%3D 10.1165/ajrcmb.10.1.8292378
    • Canonico AE, Conary JT, Meyrick BO, Brigham KL. Aerosol and intravenous transfection of human alpha 1-antitrypsin gene to lungs of rabbits. Am J Respir Cell Mol Biol. 1994;10(1):24-9.
    • (1994) Am J Respir Cell Mol Biol , vol.10 , Issue.1 , pp. 24-29
    • Canonico, A.E.1    Conary, J.T.2    Meyrick, B.O.3    Brigham, K.L.4
  • 95
    • 70349481529 scopus 로고    scopus 로고
    • Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy
    • 19706466 1:CAS:528:DC%2BD1MXht1Kqu7rO 10.1073/pnas.0904514106
    • Brantly ML, Chulay JD, Wang L, Mueller C, Humphries M, Spencer LT, et al. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proc Natl Acad Sci USA. 2009;106(38):16363-8.
    • (2009) Proc Natl Acad Sci USA , vol.106 , Issue.38 , pp. 16363-16368
    • Brantly, M.L.1    Chulay, J.D.2    Wang, L.3    Mueller, C.4    Humphries, M.5    Spencer, L.T.6
  • 96
    • 80155145248 scopus 로고    scopus 로고
    • Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing alpha1-antitrypsin: Interim results
    • 21609134 1:CAS:528:DC%2BC3MXhsVSitbnN 10.1089/hum.2011.053
    • Flotte TR, Trapnell BC, Humphries M, Carey B, Calcedo R, Rouhani F, et al. Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing alpha1-antitrypsin: interim results. Hum Gene Ther. 2011;22(10):1239-47.
    • (2011) Hum Gene Ther , vol.22 , Issue.10 , pp. 1239-1247
    • Flotte, T.R.1    Trapnell, B.C.2    Humphries, M.3    Carey, B.4    Calcedo, R.5    Rouhani, F.6
  • 97
    • 28844483864 scopus 로고    scopus 로고
    • High levels of persistent expression of α1-Antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses
    • DOI 10.1016/j.ymthe.2005.09.003, PII S1525001605015911
    • De BP, Heguy A, Hackett NR, Ferris B, Leopold PL, Lee J, et al. High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses. Mol Ther. 2006;13(1):67-76. (Pubitemid 41763484)
    • (2006) Molecular Therapy , vol.13 , Issue.1 , pp. 67-76
    • De, B.P.1    Heguy, A.2    Hackett, N.R.3    Ferris, B.4    Leopold, P.L.5    Lee, J.6    Pierre, L.7    Gao, G.8    Wilson, J.M.9    Crystal, R.G.10
  • 98
    • 36148973187 scopus 로고    scopus 로고
    • 1-AT-deficient monocytes
    • DOI 10.1089/hum.2007.073
    • McNab GL, Ahmad A, Mistry D, Stockley RA. Modification of gene expression and increase in alpha1-antitrypsin (alpha1-AT) secretion after homologous recombination in alpha1-AT-deficient monocytes. Hum Gene Ther. 2007;18(11):1171-7. (Pubitemid 350114689)
    • (2007) Human Gene Therapy , vol.18 , Issue.11 , pp. 1171-1177
    • McNab, G.L.1    Ahmad, A.2    Mistry, D.3    Stockley, R.A.4
  • 99
    • 84862705104 scopus 로고    scopus 로고
    • A novel model and molecular therapy for Z alpha-1 antitrypsin deficiency
    • 22076419 1:CAS:528:DC%2BC38XjvFKktLY%3D 10.1007/s00335-011-9370-2
    • McNab GL, Dafforn TR, Wood A, Sapey E, Stockley RA. A novel model and molecular therapy for Z alpha-1 antitrypsin deficiency. Mamm Genome. 2012;23(3-4):241-9.
    • (2012) Mamm Genome , vol.23 , Issue.3-4 , pp. 241-249
    • McNab, G.L.1    Dafforn, T.R.2    Wood, A.3    Sapey, E.4    Stockley, R.A.5
  • 100
    • 48649084090 scopus 로고    scopus 로고
    • Sustained expression of alpha1-antitrypsin after transplantation of manipulated hematopoietic stem cells
    • 18323534 1:CAS:528:DC%2BD1cXpsFOntrc%3D 10.1165/rcmb.2007-0133OC
    • Wilson AA, Kwok LW, Hovav AH, Ohle SJ, Little FF, Fine A, et al. Sustained expression of alpha1-antitrypsin after transplantation of manipulated hematopoietic stem cells. Am J Respir Cell Mol Biol. 2008;39(2):133-41.
    • (2008) Am J Respir Cell Mol Biol , vol.39 , Issue.2 , pp. 133-141
    • Wilson, A.A.1    Kwok, L.W.2    Hovav, A.H.3    Ohle, S.J.4    Little, F.F.5    Fine, A.6
  • 101
    • 77955272990 scopus 로고    scopus 로고
    • Establishment of lentiviral-vector-mediated model of human alpha-1 antitrypsin delivery into hepatocyte-like cells differentiated from mesenchymal stem cells
    • 20434188 1:CAS:528:DC%2BC3cXntVOrsrg%3D 10.1016/j.tice.2010.03.007
    • Ghaedi M, Lotfi AS, Soleimani M. Establishment of lentiviral-vector- mediated model of human alpha-1 antitrypsin delivery into hepatocyte-like cells differentiated from mesenchymal stem cells. Tissue Cell. 2010;42(3):181-9.
    • (2010) Tissue Cell , vol.42 , Issue.3 , pp. 181-189
    • Ghaedi, M.1    Lotfi, A.S.2    Soleimani, M.3
  • 102
    • 79954910852 scopus 로고    scopus 로고
    • Adipose tissue-derived mesenchymal stem cell-based liver gene delivery
    • 21168381 1:CAS:528:DC%2BC3MXkvFGhu7o%3D 10.1016/j.jhep.2010.07.051
    • Li H, Zhang B, Lu Y, Jorgensen M, Petersen B, Song S. Adipose tissue-derived mesenchymal stem cell-based liver gene delivery. J Hepatol. 2011;54(5):930-8.
    • (2011) J Hepatol , vol.54 , Issue.5 , pp. 930-938
    • Li, H.1    Zhang, B.2    Lu, Y.3    Jorgensen, M.4    Petersen, B.5    Song, S.6
  • 103
    • 77955176432 scopus 로고    scopus 로고
    • Ex vivo transduction and transplantation of bone marrow cells for liver gene delivery of alpha1-antitrypsin
    • 20551917 1:CAS:528:DC%2BC3cXntlOht7g%3D 10.1038/mt.2010.116
    • Li H, Lu Y, Witek RP, Chang LJ, Campbell-Thompson M, Jorgensen M, et al. Ex vivo transduction and transplantation of bone marrow cells for liver gene delivery of alpha1-antitrypsin. Mol Ther. 2010;18(8):1553-8.
    • (2010) Mol Ther , vol.18 , Issue.8 , pp. 1553-1558
    • Li, H.1    Lu, Y.2    Witek, R.P.3    Chang, L.J.4    Campbell-Thompson, M.5    Jorgensen, M.6
  • 104
    • 80054987997 scopus 로고    scopus 로고
    • Targeted gene correction of alpha1-antitrypsin deficiency in induced pluripotent stem cells
    • 21993621 1:CAS:528:DC%2BC3MXhtlSkur7P 10.1038/nature10424
    • Yusa K, Rashid ST, Strick-Marchand H, Varela I, Liu PQ, Paschon DE, et al. Targeted gene correction of alpha1-antitrypsin deficiency in induced pluripotent stem cells. Nature. 2011;478(7369):391-4.
    • (2011) Nature , vol.478 , Issue.7369 , pp. 391-394
    • Yusa, K.1    Rashid, S.T.2    Strick-Marchand, H.3    Varela, I.4    Liu, P.Q.5    Paschon, D.E.6
  • 105
    • 79960617712 scopus 로고    scopus 로고
    • The fibrinogen cleavage product Aalpha-Val360, a specific marker of neutrophil elastase activity in vivo
    • 21617168 10.1136/thx.2010.154690
    • Carter RI, Mumford RA, Treonze KM, Finke PE, Davies P, Si Q, et al. The fibrinogen cleavage product Aalpha-Val360, a specific marker of neutrophil elastase activity in vivo. Thorax. 2011;66(8):686-91.
    • (2011) Thorax , vol.66 , Issue.8 , pp. 686-691
    • Carter, R.I.1    Mumford, R.A.2    Treonze, K.M.3    Finke, P.E.4    Davies, P.5    Si, Q.6
  • 106
    • 0025775006 scopus 로고
    • Purification and biochemical characterization of recombinant alpha 1-antitrypsin variants expressed in Escherichia coli
    • 2012806 1:CAS:528:DyaK3MXhsF2lsL0%3D 10.1021/bi00228a017
    • Bischoff R, Speck D, Lepage P, Delatre L, Ledoux C, Brown SW, et al. Purification and biochemical characterization of recombinant alpha 1-antitrypsin variants expressed in Escherichia coli. Biochemistry. 1991;30(14):3464-72.
    • (1991) Biochemistry , vol.30 , Issue.14 , pp. 3464-3472
    • Bischoff, R.1    Speck, D.2    Lepage, P.3    Delatre, L.4    Ledoux, C.5    Brown, S.W.6
  • 107
    • 0032439236 scopus 로고    scopus 로고
    • Protein engineering of chimeric Serpins: An investigation into effects of the serpin scaffold and reactive centre loop length
    • Bottomley SP, Stone SR. Protein engineering of chimeric Serpins: an investigation into effects of the serpin scaffold and reactive centre loop length. Protein Eng. 1998;11(12):1243-7. (Pubitemid 29012135)
    • (1998) Protein Engineering , vol.11 , Issue.12 , pp. 1243-1247
    • Bottomley, S.P.1    Stone, S.R.2
  • 111
    • 0028973156 scopus 로고
    • Purification and characterization of alpha 1-antitrypsin secreted by recombinant yeast Saccharomyces diastaticus
    • 7576538 1:CAS:528:DyaK2MXos1Gns7k%3D 10.1016/0168-1656(95)00079-6
    • Kwon KS, Song M, Yu MH. Purification and characterization of alpha 1-antitrypsin secreted by recombinant yeast Saccharomyces diastaticus. J Biotechnol. 1995;42(3):191-5.
    • (1995) J Biotechnol , vol.42 , Issue.3 , pp. 191-195
    • Kwon, K.S.1    Song, M.2    Yu, M.H.3
  • 112
    • 0035814361 scopus 로고    scopus 로고
    • 1-antitrypsin
    • DOI 10.1016/S0141-0229(01)00444-6, PII S0141022901004446
    • Tamer IM, Chisti Y. Production and recovery of recombinant protease inhibitor alpha 1 antitrypsin. Enzyme Microb Technol. 2001;29:611-20. (Pubitemid 33132169)
    • (2001) Enzyme and Microbial Technology , vol.29 , Issue.10 , pp. 611-620
    • Tamer, I.M.1    Chisti, Y.2
  • 113
    • 0033678797 scopus 로고    scopus 로고
    • Effects of sugar concentration on recombinant human alpha(1)-antitrypsin production by genetically engineered rice cell
    • 11080650 1:CAS:528:DC%2BD3cXoslGlt78%3D 10.1016/S1369-703X(00)00088-7
    • Terashima M, Ejiri Y, Hashikawa N, Yoshida H. Effects of sugar concentration on recombinant human alpha(1)-antitrypsin production by genetically engineered rice cell. Biochem Eng J. 2000;6(3):201-5.
    • (2000) Biochem Eng J , vol.6 , Issue.3 , pp. 201-205
    • Terashima, M.1    Ejiri, Y.2    Hashikawa, N.3    Yoshida, H.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.