The effect of neonatal gene therapy with a gamma retroviral vector on cardiac valve disease in mucopolysaccharidosis VII dogs after a decade
Mucopolysaccharidosis VII (MPS VII) is due to deficient activity of the lysosomal enzyme β-glucuronidase (GUSB) and results in the accumulation of glycosaminoglycans (GAGs). This study determined the long-term effect of neonatal intravenous injection of a gamma retroviral vector (RV) on cardiac valv...
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Published in | Molecular genetics and metabolism Vol. 110; no. 3; pp. 311 - 318 |
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Main Authors | , , , , , , , |
Format | Journal Article |
Language | English |
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Elsevier Inc
01.11.2013
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Abstract | Mucopolysaccharidosis VII (MPS VII) is due to deficient activity of the lysosomal enzyme β-glucuronidase (GUSB) and results in the accumulation of glycosaminoglycans (GAGs). This study determined the long-term effect of neonatal intravenous injection of a gamma retroviral vector (RV) on cardiac valve disease in MPS VII dogs. Transduced hepatocytes secreted GUSB into the blood for up to 11years at levels similar to or greater than those achieved with enzyme replacement therapy (ERT). Valve regurgitation and thickening were scored from 0 (normal) to +4 (severely abnormal). At 1year, untreated MPS VII dogs had mitral regurgitation, mitral valve thickening, aortic regurgitation, and aortic valve thickening scores of 2.3±0.7, 2.3±0.6, 1.8±0.5, and 1.6±0.7, respectively, which were higher than the values of 0.6±0.1, 0.1±0.4, 0.3±0.8, and 0.1±0.4, respectively, in treated MPS VII dogs. Treated MPS VII dogs maintained low aortic regurgitation and aortic valve thickening scores in their lifetime. Although mitral regurgitation and mitral valve thickening scores increased to 2.0 at ≥8years of age in the treated MPS VII dogs, older normal dogs from the colony had similar scores, making it difficult to assess mitral valve disease. Older treated dogs had calcification within the mitral and the aortic valve annulus, while GUSB staining demonstrated enzyme activity within the mitral valve. We conclude that neonatal RV-mediated gene therapy reduced cardiac valve disease in MPS VII dogs for up to 11years, and propose that neonatal initiation of ERT should have a similar effect.
•Neonatal gene therapy reduces mitral valve disease in MPS VII dogs for 7years.•Neonatal gene therapy reduces aortic valve disease in MPS VII dogs for 11years.•Results predict that initiation of ERT at birth will reduce heart disease long-term. |
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AbstractList | Mucopolysaccharidosis VII (MPS VII) is due to deficient activity of the lysosomal enzyme β-glucuronidase (GUSB) and results in the accumulation of glycosaminoglycans (GAGs). This study determined the long-term effect of neonatal intravenous injection of a gamma retroviral vector (RV) on cardiac valve disease in MPS VII dogs. Transduced hepatocytes secreted GUSB into blood for up to 11 years at levels similar to or greater than those achieved with enzyme replacement therapy (ERT). Valve regurgitation and thickening were scored from 0 (normal) to +4 (severely abnormal). At 1 year, untreated MPS VII dogs had mitral regurgitation, mitral valve thickening, aortic regurgitation, and aortic valve thickening scores of 2.3±0.7, 2.3±0.6, 1.8±0.5, and 1.6±0.7, respectively, which were higher than the values of 0.6±0.1, 0.1±0.4, 0.3±0.8, and 0.1±0.4, respectively, in treated MPS VII dogs. Treated MPS VII dogs maintained low aortic regurgitation and aortic valve thickening scores for their lifetime. Although mitral regurgitation and mitral valve thickening scores increased to 2.0 at ≥8 years of age in the treated MPS VII dogs, older normal dogs from the colony had similar scores, making it difficult to assess mitral valve disease. Older treated dogs had calcification within the mitral and aortic valve annulus, while GUSB staining demonstrated enzyme activity within the mitral valve. We conclude that neonatal RV-mediated gene therapy reduced cardiac valve disease in MPS VII dogs for up to 11 years, and propose that neonatal initiation of ERT should have a similar effect. Mucopolysaccharidosis VII (MPS VII) is due to deficient activity of the lysosomal enzyme β-glucuronidase (GUSB) and results in the accumulation of glycosaminoglycans (GAGs). This study determined the long-term effect of neonatal intravenous injection of a gamma retroviral vector (RV) on cardiac valve disease in MPS VII dogs. Transduced hepatocytes secreted GUSB into the blood for up to 11years at levels similar to or greater than those achieved with enzyme replacement therapy (ERT). Valve regurgitation and thickening were scored from 0 (normal) to +4 (severely abnormal). At 1year, untreated MPS VII dogs had mitral regurgitation, mitral valve thickening, aortic regurgitation, and aortic valve thickening scores of 2.3±0.7, 2.3±0.6, 1.8±0.5, and 1.6±0.7, respectively, which were higher than the values of 0.6±0.1, 0.1±0.4, 0.3±0.8, and 0.1±0.4, respectively, in treated MPS VII dogs. Treated MPS VII dogs maintained low aortic regurgitation and aortic valve thickening scores in their lifetime. Although mitral regurgitation and mitral valve thickening scores increased to 2.0 at ≥8years of age in the treated MPS VII dogs, older normal dogs from the colony had similar scores, making it difficult to assess mitral valve disease. Older treated dogs had calcification within the mitral and the aortic valve annulus, while GUSB staining demonstrated enzyme activity within the mitral valve. We conclude that neonatal RV-mediated gene therapy reduced cardiac valve disease in MPS VII dogs for up to 11years, and propose that neonatal initiation of ERT should have a similar effect. •Neonatal gene therapy reduces mitral valve disease in MPS VII dogs for 7years.•Neonatal gene therapy reduces aortic valve disease in MPS VII dogs for 11years.•Results predict that initiation of ERT at birth will reduce heart disease long-term. Mucopolysaccharidosis VII (MPS VII) is due to deficient activity of the lysosomal enzyme beta -glucuronidase (GUSB) and results in the accumulation of glycosaminoglycans (GAGs). This study determined the long-term effect of neonatal intravenous injection of a gamma retroviral vector (RV) on cardiac valve disease in MPS VII dogs. Transduced hepatocytes secreted GUSB into the blood for up to 11 years at levels similar to or greater than those achieved with enzyme replacement therapy (ERT). Valve regurgitation and thickening were scored from 0 (normal) to + 4 (severely abnormal). At 1 year, untreated MPS VII dogs had mitral regurgitation, mitral valve thickening, aortic regurgitation, and aortic valve thickening scores of 2.3 plus or minus 0.7, 2.3 plus or minus 0.6, 1.8 plus or minus 0.5, and 1.6 plus or minus 0.7, respectively, which were higher than the values of 0.6 plus or minus 0.1, 0.1 plus or minus 0.4, 0.3 plus or minus 0.8, and 0.1 plus or minus 0.4, respectively, in treated MPS VII dogs. Treated MPS VII dogs maintained low aortic regurgitation and aortic valve thickening scores in their lifetime. Although mitral regurgitation and mitral valve thickening scores increased to 2.0 at greater than or equal to 8 years of age in the treated MPS VII dogs, older normal dogs from the colony had similar scores, making it difficult to assess mitral valve disease. Older treated dogs had calcification within the mitral and the aortic valve annulus, while GUSB staining demonstrated enzyme activity within the mitral valve. We conclude that neonatal RV-mediated gene therapy reduced cardiac valve disease in MPS VII dogs for up to 11 years, and propose that neonatal initiation of ERT should have a similar effect. Mucopolysaccharidosis VII (MPS VII) is due to deficient activity of the lysosomal enzyme β-glucuronidase (GUSB) and results in the accumulation of glycosaminoglycans (GAGs). This study determined the long-term effect of neonatal intravenous injection of a gamma retroviral vector (RV) on cardiac valve disease in MPS VII dogs. Transduced hepatocytes secreted GUSB into the blood for up to 11 years at levels similar to or greater than those achieved with enzyme replacement therapy (ERT). Valve regurgitation and thickening were scored from 0 (normal) to +4 (severely abnormal). At 1 year, untreated MPS VII dogs had mitral regurgitation, mitral valve thickening, aortic regurgitation, and aortic valve thickening scores of 2.3 ± 0.7, 2.3 ± 0.6, 1.8 ± 0.5, and 1.6 ± 0.7, respectively, which were higher than the values of 0.6 ± 0.1, 0.1 ± 0.4, 0.3 ± 0.8, and 0.1 ± 0.4, respectively, in treated MPS VII dogs. Treated MPS VII dogs maintained low aortic regurgitation and aortic valve thickening scores in their lifetime. Although mitral regurgitation and mitral valve thickening scores increased to 2.0 at ≥ 8 years of age in the treated MPS VII dogs, older normal dogs from the colony had similar scores, making it difficult to assess mitral valve disease. Older treated dogs had calcification within the mitral and the aortic valve annulus, while GUSB staining demonstrated enzyme activity within the mitral valve. We conclude that neonatal RV-mediated gene therapy reduced cardiac valve disease in MPS VII dogs for up to 11 years, and propose that neonatal initiation of ERT should have a similar effect. |
Author | Liu, Yuli Sleeper, Meg M. Ponder, Katherine P. Bigg, Paul W. O'Donnell, Patricia A. Wu, Susan Casal, Margret L. Haskins, Mark E. |
AuthorAffiliation | 3 Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 4 Department of Biochemistry and Molecular Biophysics, Washington University School of Medicine, St. Louis MO 2 Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 1 Department of Internal Medicine, Washington University School of Medicine, St. Louis MO |
AuthorAffiliation_xml | – name: 3 Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania – name: 2 Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania – name: 1 Department of Internal Medicine, Washington University School of Medicine, St. Louis MO – name: 4 Department of Biochemistry and Molecular Biophysics, Washington University School of Medicine, St. Louis MO |
Author_xml | – sequence: 1 givenname: Paul W. surname: Bigg fullname: Bigg, Paul W. organization: Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO, USA – sequence: 2 givenname: Meg M. surname: Sleeper fullname: Sleeper, Meg M. organization: Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA – sequence: 3 givenname: Patricia A. surname: O'Donnell fullname: O'Donnell, Patricia A. organization: Department of Pathobiology, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA – sequence: 4 givenname: Yuli surname: Liu fullname: Liu, Yuli organization: Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO, USA – sequence: 5 givenname: Susan surname: Wu fullname: Wu, Susan organization: Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO, USA – sequence: 6 givenname: Margret L. surname: Casal fullname: Casal, Margret L. organization: Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA – sequence: 7 givenname: Mark E. surname: Haskins fullname: Haskins, Mark E. organization: Department of Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, USA – sequence: 8 givenname: Katherine P. surname: Ponder fullname: Ponder, Katherine P. email: kponder@dom.wustl.edu organization: Department of Internal Medicine, Washington University School of Medicine, St. Louis, MO, USA |
BackLink | https://www.ncbi.nlm.nih.gov/pubmed/23860311$$D View this record in MEDLINE/PubMed |
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Cites_doi | 10.1016/j.amjcard.2006.02.047 10.1017/S1047951110002015 10.1016/j.ymgme.2006.08.007 10.1038/sj.mt.6300201 10.1016/j.ymgme.2009.12.003 10.1007/BF01991908 10.1016/j.jpeds.2008.12.024 10.1007/8904_2012_208 10.1161/01.CIR.0000138747.82487.4B 10.1007/s10545-011-9359-8 10.1111/j.1399-0004.2009.01324.x 10.1016/S0022-3476(75)80968-1 10.1517/14712598.7.9.1333 10.1017/S1047951109003576 10.1001/jama.281.3.249 10.1016/S0002-9149(03)00909-3 10.1006/mthe.2002.0527 10.1016/S0022-3476(73)80162-3 10.2460/ajvr.1990.51.12.2054 10.1016/0002-9149(76)90468-9 10.1007/s10545-011-9444-z 10.1111/j.1399-3046.2011.01595.x 10.1016/j.jvc.2012.02.001 10.1073/pnas.192353499 10.1016/j.ymgme.2012.03.013 10.1006/geno.1997.5189 10.1016/j.cvsm.2010.03.008 10.1038/sj.bmt.1702448 10.1038/mt.2012.9 10.1007/s004310050872 10.1007/s10545-012-9481-2 10.1007/s10545-007-0586-y 10.1016/j.ymgme.2011.08.029 |
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Keywords | Retroviral vector Mucopolysaccharidosis Lysosomal storage disease Gene therapy Cardiac disease |
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References | Wippermann, Beck, Schranz, Huth, Michel-Behnke, Jüngst (bb0020) 1995; 154 Borgarelli, Haggstrom (bb0180) 2010; 40 Ponder, Haskins (bb0130) 2007; 7 Renteria, Ferrans, Roberts (bb0185) 1976; 38 Fesslová, Corti, Sersale, Rovelli, Russo, Mannarino, Butera, Parini (bb0025) 2009; 19 Dangel (bb0030) 1998; 157 Braunlin, Berry, Whitley (bb0090) 2006; 98 Sly, Quinton, McAlister, Rimoin (bb0045) 1973; 82 Arn, Wraith, Underhill (bb0060) 2009; 154 Dierenfeld, McEntee, Vogler, Vite, Chen, Passage, Le, Shah, Jens, Snella, Kline, Parkes, Ware, Moran, Fales-Williams, Wengert, Whitley, Betts, Boal, Riedesel, Gross, Ellinwood, Dickson (bb0125) 2010; 2 Beaudet, DiFerrante, Ferry, Nichols, Mullins (bb0040) 1975; 86 Braunlin, Stauffer, Peters, Bass, Berry, Hopwood, Krivit (bb0075) 2003; 92 Furujo, Kubo, Kosuga, Okuyama (bb0110) 2011; 104 Grigull, Sykora, Tenger, Bertram, Meyer-Marcotty, Hartmann, Bültmann, Beilken, Zivicnjak, Mynarek, Osthaus, Schilke, Kollewe, Lücke (bb0065) 2011; 15 Smith, Martin, O'Donnell, Wang, Elliott, Haskins, Ponder (bb0140) 2012; 107 Soliman, Timmermans, Nemes, Vletter, Wilson, ten Cate, Geleijnse (bb0035) 2007; 30 Vogler, Levy, Kyle, Sly, Williamson, Whyte (bb0050) 1994; 7 Xu, Haskins, Melniczek, Gao, Weil, O'Malley, O'Donnell, Mazrier, Ellinwood, Zweigle, Wolfe, Ponder (bb0145) 2002; 5 Braunlin, Rosenfeld, Kampmann, Johnson, Beck, Giugliani, Guffon, Ketteridge, Sá Miranda, Scarpa, Schwartz, Leão Teles, Wraith, Barrios, Dias da Silva, Kurio, Richardson, Gildengorin, Hopwood, Imperiale, Schatz, Decker, Harmatz, MPS VI Study Group (bb0105) 2013; 36 Sleeper, Fornasari, Ellinwood, Weil, Melniczek, O'Malley, Sammarco, Xu, Ponder, Haskins (bb0150) 2004; 110 Braunlin, Harmatz, Scarpa, Furlanetto, Kampmann, Loehr, Ponder, Roberts, Rosenfeld, Giugliani (bb0015) 2011; 34 Ray, Bouvet, DeSanto, Fyfe, Xu, Wolfe, Aguirre, Patterson, Haskins, Henthorn (bb0170) 1998; 48 Neufeld, Muenzer (bb0005) 2001 Ponder, O'Malley, Wang, O'Donnell, Traas, Knox, Aguirre, Ellinwood, Metcalf, Wang, Parkinson-Lawrence, Sleeper, Brooks, Hopwood, Haskins (bb0160) 2012; 20 Fox (bb0175) 2012; 14 Sammarco, Weil, Just, Weimelt, Hasson, O'Malley, Evans, Wang, Casal, Wolfe, Haskins (bb0085) 2000; 25 Metcalf, Linders, Wu, Bigg, O'Donnell, Sleeper, Whyte, Haskins, Ponder (bb0155) 2010; 99 Watanabe, Anagnostopoulos, Azakie (bb0070) 2011; 21 McGill, Inwood, Coman, Lipke, de Lore, Swiedler, Hopwood (bb0120) 2010; 77 Meikle, Hopwood, Clague, Carey (bb0010) 1999; 281 Sifuentes, Doroshow, Hoft, Mason, Walot, Diament, Okazaki, Huff, Cox, Swiedler, Kakkis (bb0100) 2007; 90 Gompf, Shull, Breider, Scott, Constantopoulos (bb0080) 1990; 51 Leal, de Paula, Morhy, Andrade, Kim (bb0115) Mar 5 2013 Ponder, Melniczek, Xu, Weil, O'Malley, O'Donnell, Knox, Aguirre, Mazrier, Ellinwood, Sleeper, Maguire, Volk, Mango, Zweigle, Wolfe, Haskins (bb0135) 2002; 99 Schroeder, Orchard, Whitley, Berry, Tolar, Miller, Braunlin (bb0055) 2013; 10 Traas, Wang, Ma, Tittiger, Schaller, O'donnell, Sleeper, Vite, Herati, Aguirre, Haskins, Ponder (bb0165) 2007; 15 Brands, Frohn-Mulder, Hagemans, Hop, Oussoren, Helbing, van der Ploeg (bb0095) 2013; 36 17011223 - Mol Genet Metab. 2007 Feb;90(2):171-80 22278137 - J Inherit Metab Dis. 2013 Mar;36(2):227-34 12232044 - Proc Natl Acad Sci U S A. 2002 Oct 1;99(20):13102-7 11829521 - Mol Ther. 2002 Feb;5(2):141-53 21123810 - Sci Transl Med. 2010 Dec 1;2(60):60ra89 19217123 - J Pediatr. 2009 Jun;154(6):859-64.e3 17519893 - Mol Ther. 2007 Aug;15(8):1423-31 15289379 - Circulation. 2004 Aug 17;110(7):815-20 7720756 - Eur J Pediatr. 1995 Feb;154(2):98-101 22510705 - Mol Genet Metab. 2012 Sep;107(1-2):145-52 21930407 - Mol Genet Metab. 2011 Dec;104(4):597-602 22669363 - J Inherit Metab Dis. 2013 Mar;36(2):385-94 2150744 - Am J Vet Res. 1990 Dec;51(12):2054-60 16860035 - Am J Cardiol. 2006 Aug 1;98(3):416-8 21262073 - Cardiol Young. 2011 Jun;21(3):349-50 21744090 - J Inherit Metab Dis. 2011 Dec;34(6):1183-97 23430808 - JIMD Rep. 2013;10:87-94 22112002 - Pediatr Transplant. 2011 Dec;15(8):861-9 22395531 - Mol Ther. 2012 May;20(5):898-907 17727324 - Expert Opin Biol Ther. 2007 Sep;7(9):1333-45 17574537 - J Inherit Metab Dis. 2007 Oct;30(5):750-7 823811 - Am J Cardiol. 1976 Oct;38(4):487-501 19195419 - Cardiol Young. 2009 Apr;19(2):170-8 9918480 - JAMA. 1999 Jan 20;281(3):249-54 14516901 - Am J Cardiol. 2003 Oct 1;92(7):882-6 4265197 - J Pediatr. 1973 Feb;82(2):249-57 8159643 - Mod Pathol. 1994 Jan;7(1):132-7 803560 - J Pediatr. 1975 Mar;86(3):388-94 19968667 - Clin Genet. 2010 May;77(5):492-8 22386587 - J Vet Cardiol. 2012 Mar;14(1):103-26 20044292 - Mol Genet Metab. 2010 Apr;99(4):396-407 23458163 - Cardiol Young. 2014 Apr;24(2):229-35 9686810 - Eur J Pediatr. 1998 Jul;157(7):534-8 20610017 - Vet Clin North Am Small Anim Pract. 2010 Jul;40(4):651-63 10871735 - Bone Marrow Transplant. 2000 Jun;25(12):1289-97 9521879 - Genomics. 1998 Mar 1;48(2):248-53 Fox (10.1016/j.ymgme.2013.06.015_bb0175) 2012; 14 Furujo (10.1016/j.ymgme.2013.06.015_bb0110) 2011; 104 Sammarco (10.1016/j.ymgme.2013.06.015_bb0085) 2000; 25 McGill (10.1016/j.ymgme.2013.06.015_bb0120) 2010; 77 Ray (10.1016/j.ymgme.2013.06.015_bb0170) 1998; 48 Schroeder (10.1016/j.ymgme.2013.06.015_bb0055) 2013; 10 Ponder (10.1016/j.ymgme.2013.06.015_bb0130) 2007; 7 Sly (10.1016/j.ymgme.2013.06.015_bb0045) 1973; 82 Xu (10.1016/j.ymgme.2013.06.015_bb0145) 2002; 5 Meikle (10.1016/j.ymgme.2013.06.015_bb0010) 1999; 281 Vogler (10.1016/j.ymgme.2013.06.015_bb0050) 1994; 7 Sifuentes (10.1016/j.ymgme.2013.06.015_bb0100) 2007; 90 Ponder (10.1016/j.ymgme.2013.06.015_bb0160) 2012; 20 Borgarelli (10.1016/j.ymgme.2013.06.015_bb0180) 2010; 40 Metcalf (10.1016/j.ymgme.2013.06.015_bb0155) 2010; 99 Arn (10.1016/j.ymgme.2013.06.015_bb0060) 2009; 154 Traas (10.1016/j.ymgme.2013.06.015_bb0165) 2007; 15 Watanabe (10.1016/j.ymgme.2013.06.015_bb0070) 2011; 21 Gompf (10.1016/j.ymgme.2013.06.015_bb0080) 1990; 51 Brands (10.1016/j.ymgme.2013.06.015_bb0095) 2013; 36 Fesslová (10.1016/j.ymgme.2013.06.015_bb0025) 2009; 19 Braunlin (10.1016/j.ymgme.2013.06.015_bb0105) 2013; 36 Dierenfeld (10.1016/j.ymgme.2013.06.015_bb0125) 2010; 2 Dangel (10.1016/j.ymgme.2013.06.015_bb0030) 1998; 157 Braunlin (10.1016/j.ymgme.2013.06.015_bb0015) 2011; 34 Smith (10.1016/j.ymgme.2013.06.015_bb0140) 2012; 107 Braunlin (10.1016/j.ymgme.2013.06.015_bb0075) 2003; 92 Soliman (10.1016/j.ymgme.2013.06.015_bb0035) 2007; 30 Grigull (10.1016/j.ymgme.2013.06.015_bb0065) 2011; 15 Ponder (10.1016/j.ymgme.2013.06.015_bb0135) 2002; 99 Wippermann (10.1016/j.ymgme.2013.06.015_bb0020) 1995; 154 Braunlin (10.1016/j.ymgme.2013.06.015_bb0090) 2006; 98 Neufeld (10.1016/j.ymgme.2013.06.015_bb0005) 2001 Beaudet (10.1016/j.ymgme.2013.06.015_bb0040) 1975; 86 Leal (10.1016/j.ymgme.2013.06.015_bb0115) 2013 Sleeper (10.1016/j.ymgme.2013.06.015_bb0150) 2004; 110 Renteria (10.1016/j.ymgme.2013.06.015_bb0185) 1976; 38 |
References_xml | – volume: 36 start-page: 385 year: 2013 end-page: 394 ident: bb0105 article-title: Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme(®)) therapy publication-title: J. Inherit. Metab. Dis. contributor: fullname: MPS VI Study Group – volume: 99 start-page: 396 year: 2010 end-page: 407 ident: bb0155 article-title: Upregulation of elastase activity in aorta in mucopolysaccharidosis I and VII dogs may be due to increased cytokine expression publication-title: Mol. Genet. Metab. contributor: fullname: Ponder – volume: 25 start-page: 1289 year: 2000 end-page: 1297 ident: bb0085 article-title: Effects of bone marrow transplantation on the cardiovascular abnormalities in canine mucopolysaccharidosis VII publication-title: Bone Marrow Transplant. contributor: fullname: Haskins – volume: 77 start-page: 492 year: 2010 end-page: 498 ident: bb0120 article-title: Enzyme replacement therapy for mucopolysaccharidosis VI from 8 publication-title: Clin. Genet. contributor: fullname: Hopwood – start-page: 3421 year: 2001 end-page: 3452 ident: bb0005 article-title: The mucopolysaccharidoses publication-title: Metabolic and Molecular Basis of Inherited Disease contributor: fullname: Muenzer – start-page: 1 year: Mar 5 2013 end-page: 7 ident: bb0115 article-title: Advantages of early replacement therapy for mucopolysaccharidosis type VI: echocardiographic follow-up of siblings publication-title: Cardiol. Young contributor: fullname: Kim – volume: 82 start-page: 249 year: 1973 end-page: 257 ident: bb0045 article-title: Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis publication-title: J. Pediatr. contributor: fullname: Rimoin – volume: 107 start-page: 145 year: 2012 end-page: 152 ident: bb0140 article-title: Effect of neonatal gene therapy on lumbar spine disease in mucopolysaccharidosis VII dogs publication-title: Mol. Genet. Metab. contributor: fullname: Ponder – volume: 157 start-page: 534 year: 1998 end-page: 538 ident: bb0030 article-title: Cardiovascular changes in children with mucopolysaccharide storage diseases and related disorders—clinical and echocardiographic findings in 64 patients publication-title: Eur. J. Pediatr. contributor: fullname: Dangel – volume: 14 start-page: 103 year: 2012 end-page: 126 ident: bb0175 article-title: Pathology of myxomatous mitral valve disease in the dog publication-title: J. Vet. Cardiol. contributor: fullname: Fox – volume: 36 start-page: 227 year: 2013 end-page: 234 ident: bb0095 article-title: Mucopolysaccharidosis: cardiologic features and effects of enzyme-replacement therapy in 24 children with MPS I, II and VI publication-title: J. Inherit. Metab. Dis. contributor: fullname: van der Ploeg – volume: 99 start-page: 13102 year: 2002 end-page: 13107 ident: bb0135 article-title: Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs publication-title: Proc. Natl. Acad. Sci. U. S. A. contributor: fullname: Haskins – volume: 34 start-page: 1183 year: 2011 end-page: 1197 ident: bb0015 article-title: Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management publication-title: J. Inherit. Metab. Dis. contributor: fullname: Giugliani – volume: 15 start-page: 861 year: 2011 end-page: 869 ident: bb0065 article-title: Variable disease progression after successful stem cell transplantation: prospective follow-up investigations in eight patients with Hurler syndrome publication-title: Pediatr. Transplant. contributor: fullname: Lücke – volume: 30 start-page: 750 year: 2007 end-page: 757 ident: bb0035 article-title: Cardiac abnormalities in adults with the attenuated form of mucopolysaccharidosis type I publication-title: J. Inherit. Metab. Dis. contributor: fullname: Geleijnse – volume: 7 start-page: 1333 year: 2007 end-page: 1345 ident: bb0130 article-title: Gene therapy for mucopolysaccharidosis publication-title: Expert. Opin. Biol. Ther. contributor: fullname: Haskins – volume: 38 start-page: 487 year: 1976 end-page: 501 ident: bb0185 article-title: The heart in the Hurler syndrome: gross, histologic and ultrastructural observations in five necropsy cases publication-title: Am. J. Cardiol. contributor: fullname: Roberts – volume: 92 start-page: 882 year: 2003 end-page: 886 ident: bb0075 article-title: Usefulness of bone marrow transplantation in the Hurler syndrome publication-title: Am. J. Cardiol. contributor: fullname: Krivit – volume: 21 start-page: 349 year: 2011 end-page: 350 ident: bb0070 article-title: Aortic stenosis in a patient with Hurler's syndrome after bone marrow transplantation publication-title: Cardiol. Young contributor: fullname: Azakie – volume: 19 start-page: 170 year: 2009 end-page: 178 ident: bb0025 article-title: The natural course and the impact of therapies of cardiac involvement in the mucopolysaccharidoses publication-title: Cardiol. Young contributor: fullname: Parini – volume: 104 start-page: 597 year: 2011 end-page: 602 ident: bb0110 article-title: Enzyme replacement therapy attenuates disease progression in two Japanese siblings with mucopolysaccharidosis type VI publication-title: Mol. Genet. Metab. contributor: fullname: Okuyama – volume: 15 start-page: 1423 year: 2007 end-page: 1431 ident: bb0165 article-title: Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy publication-title: Mol. Ther. contributor: fullname: Ponder – volume: 281 start-page: 249 year: 1999 end-page: 254 ident: bb0010 article-title: Prevalence of lysosomal storage disorders publication-title: JAMA contributor: fullname: Carey – volume: 10 start-page: 87 year: 2013 end-page: 94 ident: bb0055 article-title: Cardiac ultrasound findings in infants with severe (Hurler phenotype) untreated mucopolysaccharidosis (MPS) type I publication-title: JIMD Rep. contributor: fullname: Braunlin – volume: 20 start-page: 898 year: 2012 end-page: 907 ident: bb0160 article-title: Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats publication-title: Mol. Ther. contributor: fullname: Haskins – volume: 40 start-page: 651 year: 2010 end-page: 663 ident: bb0180 article-title: Canine degenerative myxomatous mitral valve disease: natural history, clinical presentation and therapy publication-title: Vet. Clin. North Am. Small Anim. Pract. contributor: fullname: Haggstrom – volume: 154 start-page: 98 year: 1995 end-page: 101 ident: bb0020 article-title: Mitral and aortic regurgitation in 84 patients with mucopolysaccharidoses publication-title: Eur. J. Pediatr. contributor: fullname: Jüngst – volume: 110 start-page: 815 year: 2004 end-page: 820 ident: bb0150 article-title: Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII publication-title: Circulation contributor: fullname: Haskins – volume: 7 start-page: 132 year: 1994 end-page: 137 ident: bb0050 article-title: Mucopolysaccharidosis VII: postmortem biochemical and pathological findings in a young adult with beta-glucuronidase deficiency publication-title: Mod. Pathol. contributor: fullname: Whyte – volume: 51 start-page: 2054 year: 1990 end-page: 2060 ident: bb0080 article-title: Cardiovascular changes after bone marrow transplantation in dogs with mucopolysaccharidosis I publication-title: Am. J. Vet. Res. contributor: fullname: Constantopoulos – volume: 2 start-page: 60ra89 year: 2010 ident: bb0125 article-title: Replacing the enzyme alpha- publication-title: Sci. Total. Environ. contributor: fullname: Dickson – volume: 90 start-page: 171 year: 2007 end-page: 180 ident: bb0100 article-title: A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 publication-title: Mol. Genet. Metab. contributor: fullname: Kakkis – volume: 98 start-page: 416 year: 2006 end-page: 418 ident: bb0090 article-title: Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I publication-title: Am. J. Cardiol. contributor: fullname: Whitley – volume: 154 start-page: 859 year: 2009 end-page: 864 ident: bb0060 article-title: Characterization of surgical procedures in patients with mucopolysaccharidosis type I: findings from the MPS I Registry publication-title: J. Pediatr. contributor: fullname: Underhill – volume: 5 start-page: 141 year: 2002 end-page: 153 ident: bb0145 article-title: Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs publication-title: Mol. Ther. contributor: fullname: Ponder – volume: 86 start-page: 388 year: 1975 end-page: 394 ident: bb0040 article-title: Variation in the phenotypic expression of beta-glucuronidase deficiency publication-title: J. Pediatr. contributor: fullname: Mullins – volume: 48 start-page: 248 year: 1998 end-page: 253 ident: bb0170 article-title: Cloning of the canine beta-glucuronidase cDNA, mutation identification in canine MPS VII, and retroviral vector-mediated correction of MPS VII cells publication-title: Genomics contributor: fullname: Henthorn – volume: 98 start-page: 416 year: 2006 ident: 10.1016/j.ymgme.2013.06.015_bb0090 article-title: Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I publication-title: Am. J. Cardiol. doi: 10.1016/j.amjcard.2006.02.047 contributor: fullname: Braunlin – volume: 21 start-page: 349 year: 2011 ident: 10.1016/j.ymgme.2013.06.015_bb0070 article-title: Aortic stenosis in a patient with Hurler's syndrome after bone marrow transplantation publication-title: Cardiol. Young doi: 10.1017/S1047951110002015 contributor: fullname: Watanabe – volume: 90 start-page: 171 year: 2007 ident: 10.1016/j.ymgme.2013.06.015_bb0100 article-title: A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6years publication-title: Mol. Genet. Metab. doi: 10.1016/j.ymgme.2006.08.007 contributor: fullname: Sifuentes – volume: 15 start-page: 1423 year: 2007 ident: 10.1016/j.ymgme.2013.06.015_bb0165 article-title: Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy publication-title: Mol. Ther. doi: 10.1038/sj.mt.6300201 contributor: fullname: Traas – start-page: 3421 year: 2001 ident: 10.1016/j.ymgme.2013.06.015_bb0005 article-title: The mucopolysaccharidoses contributor: fullname: Neufeld – volume: 99 start-page: 396 year: 2010 ident: 10.1016/j.ymgme.2013.06.015_bb0155 article-title: Upregulation of elastase activity in aorta in mucopolysaccharidosis I and VII dogs may be due to increased cytokine expression publication-title: Mol. Genet. Metab. doi: 10.1016/j.ymgme.2009.12.003 contributor: fullname: Metcalf – volume: 154 start-page: 98 year: 1995 ident: 10.1016/j.ymgme.2013.06.015_bb0020 article-title: Mitral and aortic regurgitation in 84 patients with mucopolysaccharidoses publication-title: Eur. J. Pediatr. doi: 10.1007/BF01991908 contributor: fullname: Wippermann – volume: 154 start-page: 859 year: 2009 ident: 10.1016/j.ymgme.2013.06.015_bb0060 article-title: Characterization of surgical procedures in patients with mucopolysaccharidosis type I: findings from the MPS I Registry publication-title: J. Pediatr. doi: 10.1016/j.jpeds.2008.12.024 contributor: fullname: Arn – volume: 10 start-page: 87 year: 2013 ident: 10.1016/j.ymgme.2013.06.015_bb0055 article-title: Cardiac ultrasound findings in infants with severe (Hurler phenotype) untreated mucopolysaccharidosis (MPS) type I publication-title: JIMD Rep. doi: 10.1007/8904_2012_208 contributor: fullname: Schroeder – volume: 110 start-page: 815 year: 2004 ident: 10.1016/j.ymgme.2013.06.015_bb0150 article-title: Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII publication-title: Circulation doi: 10.1161/01.CIR.0000138747.82487.4B contributor: fullname: Sleeper – volume: 34 start-page: 1183 year: 2011 ident: 10.1016/j.ymgme.2013.06.015_bb0015 article-title: Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management publication-title: J. Inherit. Metab. Dis. doi: 10.1007/s10545-011-9359-8 contributor: fullname: Braunlin – volume: 77 start-page: 492 year: 2010 ident: 10.1016/j.ymgme.2013.06.015_bb0120 article-title: Enzyme replacement therapy for mucopolysaccharidosis VI from 8weeks of age—a sibling control study publication-title: Clin. Genet. doi: 10.1111/j.1399-0004.2009.01324.x contributor: fullname: McGill – volume: 86 start-page: 388 year: 1975 ident: 10.1016/j.ymgme.2013.06.015_bb0040 article-title: Variation in the phenotypic expression of beta-glucuronidase deficiency publication-title: J. Pediatr. doi: 10.1016/S0022-3476(75)80968-1 contributor: fullname: Beaudet – volume: 7 start-page: 1333 year: 2007 ident: 10.1016/j.ymgme.2013.06.015_bb0130 article-title: Gene therapy for mucopolysaccharidosis publication-title: Expert. Opin. Biol. Ther. doi: 10.1517/14712598.7.9.1333 contributor: fullname: Ponder – volume: 19 start-page: 170 year: 2009 ident: 10.1016/j.ymgme.2013.06.015_bb0025 article-title: The natural course and the impact of therapies of cardiac involvement in the mucopolysaccharidoses publication-title: Cardiol. Young doi: 10.1017/S1047951109003576 contributor: fullname: Fesslová – volume: 281 start-page: 249 year: 1999 ident: 10.1016/j.ymgme.2013.06.015_bb0010 article-title: Prevalence of lysosomal storage disorders publication-title: JAMA doi: 10.1001/jama.281.3.249 contributor: fullname: Meikle – volume: 92 start-page: 882 year: 2003 ident: 10.1016/j.ymgme.2013.06.015_bb0075 article-title: Usefulness of bone marrow transplantation in the Hurler syndrome publication-title: Am. J. Cardiol. doi: 10.1016/S0002-9149(03)00909-3 contributor: fullname: Braunlin – volume: 5 start-page: 141 year: 2002 ident: 10.1016/j.ymgme.2013.06.015_bb0145 article-title: Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs publication-title: Mol. Ther. doi: 10.1006/mthe.2002.0527 contributor: fullname: Xu – volume: 82 start-page: 249 year: 1973 ident: 10.1016/j.ymgme.2013.06.015_bb0045 article-title: Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis publication-title: J. Pediatr. doi: 10.1016/S0022-3476(73)80162-3 contributor: fullname: Sly – volume: 51 start-page: 2054 year: 1990 ident: 10.1016/j.ymgme.2013.06.015_bb0080 article-title: Cardiovascular changes after bone marrow transplantation in dogs with mucopolysaccharidosis I publication-title: Am. J. Vet. Res. doi: 10.2460/ajvr.1990.51.12.2054 contributor: fullname: Gompf – volume: 38 start-page: 487 year: 1976 ident: 10.1016/j.ymgme.2013.06.015_bb0185 article-title: The heart in the Hurler syndrome: gross, histologic and ultrastructural observations in five necropsy cases publication-title: Am. J. Cardiol. doi: 10.1016/0002-9149(76)90468-9 contributor: fullname: Renteria – volume: 36 start-page: 227 year: 2013 ident: 10.1016/j.ymgme.2013.06.015_bb0095 article-title: Mucopolysaccharidosis: cardiologic features and effects of enzyme-replacement therapy in 24 children with MPS I, II and VI publication-title: J. Inherit. Metab. Dis. doi: 10.1007/s10545-011-9444-z contributor: fullname: Brands – volume: 2 start-page: 60ra89 year: 2010 ident: 10.1016/j.ymgme.2013.06.015_bb0125 article-title: Replacing the enzyme alpha-l-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I publication-title: Sci. Total. Environ. contributor: fullname: Dierenfeld – volume: 15 start-page: 861 year: 2011 ident: 10.1016/j.ymgme.2013.06.015_bb0065 article-title: Variable disease progression after successful stem cell transplantation: prospective follow-up investigations in eight patients with Hurler syndrome publication-title: Pediatr. Transplant. doi: 10.1111/j.1399-3046.2011.01595.x contributor: fullname: Grigull – volume: 14 start-page: 103 year: 2012 ident: 10.1016/j.ymgme.2013.06.015_bb0175 article-title: Pathology of myxomatous mitral valve disease in the dog publication-title: J. Vet. Cardiol. doi: 10.1016/j.jvc.2012.02.001 contributor: fullname: Fox – volume: 99 start-page: 13102 year: 2002 ident: 10.1016/j.ymgme.2013.06.015_bb0135 article-title: Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs publication-title: Proc. Natl. Acad. Sci. U. S. A. doi: 10.1073/pnas.192353499 contributor: fullname: Ponder – volume: 107 start-page: 145 year: 2012 ident: 10.1016/j.ymgme.2013.06.015_bb0140 article-title: Effect of neonatal gene therapy on lumbar spine disease in mucopolysaccharidosis VII dogs publication-title: Mol. Genet. Metab. doi: 10.1016/j.ymgme.2012.03.013 contributor: fullname: Smith – volume: 48 start-page: 248 year: 1998 ident: 10.1016/j.ymgme.2013.06.015_bb0170 article-title: Cloning of the canine beta-glucuronidase cDNA, mutation identification in canine MPS VII, and retroviral vector-mediated correction of MPS VII cells publication-title: Genomics doi: 10.1006/geno.1997.5189 contributor: fullname: Ray – volume: 40 start-page: 651 year: 2010 ident: 10.1016/j.ymgme.2013.06.015_bb0180 article-title: Canine degenerative myxomatous mitral valve disease: natural history, clinical presentation and therapy publication-title: Vet. Clin. North Am. Small Anim. Pract. doi: 10.1016/j.cvsm.2010.03.008 contributor: fullname: Borgarelli – volume: 25 start-page: 1289 year: 2000 ident: 10.1016/j.ymgme.2013.06.015_bb0085 article-title: Effects of bone marrow transplantation on the cardiovascular abnormalities in canine mucopolysaccharidosis VII publication-title: Bone Marrow Transplant. doi: 10.1038/sj.bmt.1702448 contributor: fullname: Sammarco – volume: 20 start-page: 898 year: 2012 ident: 10.1016/j.ymgme.2013.06.015_bb0160 article-title: Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats publication-title: Mol. Ther. doi: 10.1038/mt.2012.9 contributor: fullname: Ponder – volume: 157 start-page: 534 year: 1998 ident: 10.1016/j.ymgme.2013.06.015_bb0030 article-title: Cardiovascular changes in children with mucopolysaccharide storage diseases and related disorders—clinical and echocardiographic findings in 64 patients publication-title: Eur. J. Pediatr. doi: 10.1007/s004310050872 contributor: fullname: Dangel – volume: 36 start-page: 385 year: 2013 ident: 10.1016/j.ymgme.2013.06.015_bb0105 article-title: Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme(®)) therapy publication-title: J. Inherit. Metab. Dis. doi: 10.1007/s10545-012-9481-2 contributor: fullname: Braunlin – volume: 30 start-page: 750 year: 2007 ident: 10.1016/j.ymgme.2013.06.015_bb0035 article-title: Cardiac abnormalities in adults with the attenuated form of mucopolysaccharidosis type I publication-title: J. Inherit. Metab. Dis. doi: 10.1007/s10545-007-0586-y contributor: fullname: Soliman – volume: 104 start-page: 597 year: 2011 ident: 10.1016/j.ymgme.2013.06.015_bb0110 article-title: Enzyme replacement therapy attenuates disease progression in two Japanese siblings with mucopolysaccharidosis type VI publication-title: Mol. Genet. Metab. doi: 10.1016/j.ymgme.2011.08.029 contributor: fullname: Furujo – start-page: 1 year: 2013 ident: 10.1016/j.ymgme.2013.06.015_bb0115 article-title: Advantages of early replacement therapy for mucopolysaccharidosis type VI: echocardiographic follow-up of siblings publication-title: Cardiol. Young contributor: fullname: Leal – volume: 7 start-page: 132 year: 1994 ident: 10.1016/j.ymgme.2013.06.015_bb0050 article-title: Mucopolysaccharidosis VII: postmortem biochemical and pathological findings in a young adult with beta-glucuronidase deficiency publication-title: Mod. Pathol. contributor: fullname: Vogler |
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Snippet | Mucopolysaccharidosis VII (MPS VII) is due to deficient activity of the lysosomal enzyme β-glucuronidase (GUSB) and results in the accumulation of... Mucopolysaccharidosis VII (MPS VII) is due to deficient activity of the lysosomal enzyme beta -glucuronidase (GUSB) and results in the accumulation of... |
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StartPage | 311 |
SubjectTerms | Animals Animals, Newborn Aortic Valve - diagnostic imaging Aortic Valve - pathology Cardiac disease Disease Models, Animal Dogs Echocardiography Female Gammaretrovirus - genetics Gene therapy Genetic Therapy Genetic Vectors - genetics Glucuronidase - genetics Glucuronidase - metabolism Heart Valve Diseases - diagnostic imaging Heart Valve Diseases - etiology Heart Valve Diseases - therapy Lysosomal storage disease Male Mitral Valve - diagnostic imaging Mitral Valve - pathology Mucopolysaccharidosis Mucopolysaccharidosis VII - complications Mucopolysaccharidosis VII - genetics Retroviral vector |
Title | The effect of neonatal gene therapy with a gamma retroviral vector on cardiac valve disease in mucopolysaccharidosis VII dogs after a decade |
URI | https://dx.doi.org/10.1016/j.ymgme.2013.06.015 https://www.ncbi.nlm.nih.gov/pubmed/23860311 https://search.proquest.com/docview/1443423686 https://search.proquest.com/docview/1516750153 https://pubmed.ncbi.nlm.nih.gov/PMC3800273 |
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