Serum MIP-1 α level: A biomarker for the follow-up of lentiviral therapy in mucopolysaccharidosis IIIB mice

Journal of Inherited Metabolic Disease

Volumn 33, Issue 2, 2010, Pages 159-165

  Di Natale, Paola ^a   Di Domenico, Carmela ^a   Di Napoli, Daniele ^b  

^a UNIVERSITY OF NAPLES FEDERICO II   (Italy)

^b CARDARELLI HOSPITAL   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA N ACETYLGLUCOSAMINIDASE; BIOLOGICAL MARKER; BRAIN DERIVED NEUROTROPHIC FACTOR; GLYCOSAMINOGLYCAN; LENTIVIRUS VECTOR; MACROPHAGE INFLAMMATORY PROTEIN 1ALPHA;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BRAIN TISSUE; CONTROLLED STUDY; DISEASE COURSE; DISEASE SEVERITY; DRUG EFFICACY; MOUSE; MUCOPOLYSACCHARIDOSIS; NONHUMAN; PROTEIN ANALYSIS; PROTEIN BLOOD LEVEL; SANFILIPPO SYNDROME TYPE B; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ACETYLGLUCOSAMINIDASE; ANIMALS; BIOLOGICAL MARKERS; BRAIN; CHEMOKINE CCL3; DISEASE MODELS, ANIMAL; DISEASE PROGRESSION; FEMALE; FOLLOW-UP STUDIES; GENE THERAPY; LENTIVIRUS; MALE; MICE; MICE, MUTANT STRAINS; MUCOPOLYSACCHARIDOSIS III; TRANSGENES;

EID: 77953232475     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-010-9051-4     Document Type: Article

References (30)

1. J Ausseil N Desmaris S Bigou R Attali S Corbineau S Vitry M Parent D Cheillan M Fuller I Maire MT Vanier JM Heard 2008 Early neurodegeneration progresses independently of microglial activation by heparan sulfate in the brain of mucopolysaccharidosis IIIB mice PLoS ONE 3 5 e2296 10.1371/journal. pone.0002296 18509511
2. CE Beesley EP Young N Finnegan M Jackson K Mills A Vellodi M Cleary BG Winchester 2009 Discovery of a new biomarker for the mucopolysaccharidoses (MPS), dipeptidyl peptidase IV (DPP-IV, CD26), by SELDI-TOF mass spectrometry Mol Genet Metab 96 218 224 10.1016/j.ymgme.2008.12.002 1:CAS:528: DC%2BD1MXjt1agur0%3D 19153055
3. RG Boot M Verhoek M de Fost CEM Hollak M Maas B Bleijlevens MJ van Breemen M van Meurs LA Boven JD Laman MT Moran TM Cox JMFG Aerts 2004 Marked elevation of the chemokine CCL18/PARC in Gaucher disease: a novel surrogate marker for assessing tharepeutic intervation Blood 103 33 39 10.1182/blood-2003-05-1612 1:CAS:528:DC%2BD2cXhsFCrug%3D%3D 12969956 (Pubitemid 38029915)
4. R Christensen AB Marcussen G Wörtwein GM Knudsen S Aznar 2008 Abeta (1-42) injection causes memory impairment, lowered cortical and serum BDNF levels, and decreased hippocampal 5-HT(2A) levels Exp Neurol 210 164 171 10.1016/j.expneurol.2007.10.009 1:CAS:528:DC%2BD1cXisVWis7Y%3D 18053988 (Pubitemid 351258077)
5. A Cressant N Desmaris L Veot T Brejot R Froissart MT Vanier I Maire JM Herad 2004 Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-assciated virus-mediated gene transfer in the striatun J Neurosci 24 10229 10239 10.1523/JNEUROSCI.3558-04.2004 1:CAS:528:DC%2BD2cXhtVeqtb%2FN 15537895 (Pubitemid 39492050)
6. R Dhami MA Passini EH Schuchman 2006 Identification of novel biomarkers for Niemann-Pick disease using gene expression analysis of acid sphingomyelinase knockout mice Mol Ther 13 556 564 10.1016/j.ymthe.2005.08.020 1:CAS:528:DC%2BD28Xhslykt7o%3D 16214420 (Pubitemid 43257623)
7. C Di Domenico GRD Villani D Di Napoli E Nusco G Calì L Nitsch P Di Natale 2009 Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB Am J Med Genet Part A 149 1209 1218
8. P Di Natale C Di Domenico N Gargiulo S Castaldo Y Gonzalez E Reyero P Mithbaokar M De Felice A Follenzi L Naldini GRD Villani 2005 Treatment of the mouse model of mucopolysaccharidosis type IIIB with lentiviral-NAGLU vector Biochem J 388 639 646 10.1042/BJ20041702 15649123 (Pubitemid 40839942)
9. P Di Natale GRD Villani R Parini M Scarpa G Parenti G Pontarelli M Grosso G Sersale R Tomanin M Sibilio R Barone A Fiumara 2008 Molecular markers for the follow-up of enzyme-replacement therapy in mucopolysaccharidosis type VI disease Biotechnol Appl Biochem 49 219 223 10.1042/BA20070093 17672828 (Pubitemid 351579074)
10. J DiRosario E Divers C Wang J Etter A Charrier P Jukkola H Auer V Best DL Newsom DM McCarty H Fu 2009 Innate and adaptive immune activation in the brain of MPS IIIB mouse model J Neurosci Res 87 4 978 990 10.1002/jnr.21912 1:CAS:528:DC%2BD1MXivVamtbs%3D 18951493
11. PS DiStefano B Friedman C Radzejewski C Alexander P Boland CM Schick RM Lindsay SJ Wiegand 1992 The neurotrophins BDNF, NT-3 And NGF display distinct patterns of retrograde axonal transport in peripheral and central neurons Neuron 8 983 993 10.1016/0896-6273(92)90213-W 1:CAS:528:DyaK38Xks1eksr0%3D 1375039
12. H Fu L Kang JS Jennings SS Moy A Perez J DiRosario DM Mccarty J Muenzer 2007 Significanlty increased lifespan and improved behavioral performances by rAVV gene delivery in adult mucopolysaccharidosis IIIB mice Gene Ther 14 1065 1077 10.1038/sj.gt.3302961 1:CAS:528:DC%2BD2sXntFyms7k%3D 17460717 (Pubitemid 47029490)
13. P Harmatz WG Kramer JJ Hopwood J Simon E Butensky SJ Swiedler 2005 Pharmacokinetic profile of recombinant human N-acetylgalactosamine 4-sulphatase enzyme replacement therapy in patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): a phase I/II study Acta Paediatr Suppl 94 61 68 10.1080/08035320510028139 1:STN:280:DC%2BD2M3lsFSiug%3D%3D 15895715
14. CEM Hollak S van Weely MHJ van Oers JMFG Aerts 1994 Marked elevation of plasma chitotriosidase activity. A novel hallmark of Gaucher disease J Clin Invest 93 1288 1292 10.1172/JCI117084 1:STN:280:DyaK2c7nvFCkug%3D%3D 8132768
15. HH Li WH Yu N Rozengurt HZ Zhao KM Lyons S Anagnostaras MS Fanselow K Suzuki MT Vanier EF Neufeld 1999 Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding alpha-N- acetylglucosaminidase Proc Natl Acad Sci USA 96 14505 14510 10.1073/pnas.96.25. 14505 1:CAS:528:DyaK1MXnvFKks7w%3D 10588735 (Pubitemid 30000680)
16. J Marsh AH Fensom 1985 4-Methylumbelliferyl alpha-N-acetylglucosaminidase activity for diagnosis of Sanfilippo B disease Clin Genet 27 258 262 1:STN:280:DyaL2M7nslKhsw%3D%3D 3921297
17. DM McCarty J DiRosario K Gulaid J Muenzer H Fu 2009 Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice Gene Ther 16 1340 1352 10.1038/gt.2009.85 1:CAS:528:DC%2BD1MXotlSgtLs%3D 19587708
18. R McGlynn K Dobrenis SU Walkley 2004 Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine model of mucopolysaccharide storage disorders J Comp Neurol 480 415 426 10.1002/cne.20355 1:CAS:528:DC%2BD2MXit1SmtA%3D%3D 15558784 (Pubitemid 39587233)
19. J Muenzer M Gucsavas-Calikoglu SE McCandless TJ Schuetz A Kimura 2007 A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome) Mol Genet Metab 90 329 337 10.1016/j.ymgme.2006.09.001 1:CAS:528:DC%2BD2sXitFOqs7k%3D 17185020 (Pubitemid 46241902)
20. Neufeld EF, Muenzer J (2001) The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds) The metabolic and molecular bases of inherited disease, 8th edn. Mc Graw-Hill, New York, pp 3421-3452
21. K Ohmi DS Greenberg KS Rajavel S Ryazantsev HH Li EF Neufeld 2003 Activated microglia in cortex of mouse models of mucopolysaccharidosis I and IIIB Proc Natl Acad Sci USA 100 1902 1907 10.1073/pnas.252784899 1:CAS:528:DC%2BD3sXhsFGrs78%3D 12576554 (Pubitemid 36254546)
22. K Ohmi LC Kudo S Ryazantsev HZ Zhao SL Karsten EF Neufeld 2009 Sanfilippo syndrome type B, a lysosomal storage disease, is also a tauopathy Proc Natl Acad Sci USA 106 8332 8337 10.1073/pnas.0903223106 1:CAS:528: DC%2BD1MXmslyksLY%3D 19416848 Epub 2009 May 5
23. W Pan WA Banks AJ Kastin 1998 Permeability of the blood- brain barrier to neurotrophins Brain Res 788 87 94 10.1016/S0006-8993(97)01525-4 1:CAS:528:DyaK1cXhvVSjurg%3D 9554964 (Pubitemid 28202147)
24. DR Randall GB Sinclair KE Colobong E Hetty LA Clarke 2006 Heparin cofactor II-thrombin complex in MPS I: a biomarker of MPS disease Mol Genet Metab 88 235 243 10.1016/j.ymgme.2006.01.005 1:CAS:528:DC%2BD28Xms1agu7c%3D 16497528 (Pubitemid 43963434)
25. DR Randall KE Colobong H Hemmelgarn GB Sinclair E Hetty A Thomas OA Bodamer B Volkmar PM Fernhoff R Casey AK Chan G Mitchell S Stockler S Melancon T Rupar LA Clarke 2008 Heparin cofactor II-thrombin complex: a biomarker of MPS disease Mol Genet Metab 94 456 461 10.1016/j.ymgme.2008.05.001 1:CAS:528:DC%2BD1cXosVOnur0%3D 18511319
26. S Ryazantsev WH Yu HZ Zhao EF Neufeld K Ohmi 2007 Lysosomal accumulation of SCMAS (subunit c of mitochondrial ATP synthase) in neurons of the mouse model of mucopolysaccharidosis IIIB Mol Genet Metab 90 393 401 10.1016/j.ymgme.2006. 11.006 1:CAS:528:DC%2BD2sXjs1Ontr4%3D 17185018 (Pubitemid 46484425)
27. GRD Villani R Faraonio S Castaldo Y Gonzalez E Reyero P Di Natale 2007 Cytokines, neurotrophins, and oxidative stress in brain disease from mucopolysaccharidosis IIIB J Neurosci Res 85 612 622 10.1002/jnr.21134 1:CAS:528:DC%2BD2sXjtFOksrk%3D 17139681
28. GRD Villani C Di Domenico A Musella F Cecere D Di Napoli P Di Natale 2009 Mucopolysaccharidosis IIIB: oxidative damage and cytotoxic cell involvement in the neuronal pathogenesis Brain Res 1279 99 108 10.1016/j.brainres.2009.03.071 1:CAS:528:DC%2BD1MXnsFajt7k%3D 19409882
29. JE Wraith LA Clarke M Beck EH Kolodny GM Pastores J Muenzer DM Rapoport KI Berger SJ Swiedler ED Kakkis T Braakman E Chadbourne K Walton-Bowen GF Cox 2004 Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase) J Pediatr 144 581 588 10.1016/j.jpeds.2004.01. 046 1:CAS:528:DC%2BD2cXjs1Wnsbg%3D 15126990 (Pubitemid 38594757)
30. Y Zheng S Ryazantsev K Ohmi HZ Zhao N Rozengurt DB Kohn EF Neufeld 2004 Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB Mol Genet Metab 82 286 295 10.1016/j.ymgme.2004.06.004 1:CAS:528:DC%2BD2cXmslOgtLc%3D 15308126 (Pubitemid 39078708)