Development of Substrate Degradation Enzyme Therapy for Mucopolysaccharidosis IVA Murine Model.
Animals
Biomarkers
Disease Models, Animal
Enzyme Replacement Therapy
Enzyme Stability
Glycosaminoglycans
/ metabolism
Glycoside Hydrolases
/ administration & dosage
Male
Mice
Mice, Knockout
Mucopolysaccharidosis IV
/ enzymology
Recombinant Proteins
Substrate Specificity
Temperature
Treatment Outcome
GALNS
MPS IVA
keratan sulfate
skeletal dysplasia
thermostable keratanase
Journal
International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791
Informations de publication
Date de publication:
24 Aug 2019
24 Aug 2019
Historique:
received:
22
07
2019
revised:
18
08
2019
accepted:
21
08
2019
entrez:
28
8
2019
pubmed:
28
8
2019
medline:
25
1
2020
Statut:
epublish
Résumé
Mucopolysaccharidosis IVA (MPS IVA) is caused by a deficiency of the lysosomal enzyme N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Conventional enzyme replacement therapy (ERT) is approved for MPS IVA. However, the fact that the infused enzyme cannot penetrate avascular lesions in cartilage leads to minimal impact on the bone lesion. Moreover, short half-life, high cost, instability, and narrow optimal pH range remain unmet challenges in ERT. Thermostable keratanase, endo-β-
Identifiants
pubmed: 31450640
pii: ijms20174139
doi: 10.3390/ijms20174139
pmc: PMC6747109
pii:
doi:
Substances chimiques
Biomarkers
0
Glycosaminoglycans
0
Recombinant Proteins
0
Glycoside Hydrolases
EC 3.2.1.-
keratan-sulfate endo-1,4-beta-galactosidase
EC 3.2.1.103
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : National MPS Society Research Grant
ID : NA
Organisme : NIH HHS
ID : P30GM114736
Pays : United States
Références
J Immunol. 2002 May 15;168(10):5233-9
pubmed: 11994480
Biochem Med. 1975 Apr;12(4):331-9
pubmed: 126060
J Biol Chem. 2003 Jul 11;278(28):25766-72
pubmed: 12732618
Hum Mol Genet. 2003 Dec 15;12(24):3349-58
pubmed: 14583446
Pediatr Res. 2004 Apr;55(4):592-7
pubmed: 14711889
J Biol Chem. 1949 Nov;181(1):149-51
pubmed: 15390401
Hum Mol Genet. 2005 Nov 15;14(22):3321-35
pubmed: 16219627
Biomed Chromatogr. 2007 Apr;21(4):356-62
pubmed: 17236248
J Inherit Metab Dis. 2007 Apr;30(2):165-74
pubmed: 17347914
J Biol Chem. 2007 Jun 22;282(25):18265-75
pubmed: 17400552
Osteoarthritis Cartilage. 2007 Nov;15(11):1311-7
pubmed: 17548214
Anal Biochem. 2007 Sep 1;368(1):79-86
pubmed: 17603992
Hum Mol Genet. 2008 Mar 15;17(6):815-24
pubmed: 18056156
J Biol Chem. 2008 Nov 7;283(45):31237-45
pubmed: 18757372
Mol Genet Metab. 2009 Jul;97(3):196-201
pubmed: 19394256
Proc Natl Acad Sci U S A. 2010 Jan 5;107(1):222-7
pubmed: 20018674
J Inherit Metab Dis. 2010 Dec;33 Suppl 3:S35-42
pubmed: 20107903
Mol Ther. 2010 Jun;18(6):1094-102
pubmed: 20332769
Med Hypotheses. 2010 Dec;75(6):642-4
pubmed: 20800368
Biomark Insights. 2011;6:69-78
pubmed: 21792275
J Neurosci. 2011 Nov 23;31(47):17091-102
pubmed: 22114278
Res Rep Endocr Disord. 2012 Dec;2012(2):65-77
pubmed: 24839594
JIMD Rep. 2014;16:15-24
pubmed: 24850234
Mol Genet Metab. 2015 Feb;114(2):195-202
pubmed: 24953405
PLoS One. 2014 Jun 25;9(6):e100882
pubmed: 24964042
Clin Pharmacokinet. 2014 Dec;53(12):1137-47
pubmed: 25234648
Mol Genet Metab. 2015 Feb;114(2):94-109
pubmed: 25537451
Mol Genet Metab Rep. 2014;1:31-41
pubmed: 25593792
JIMD Rep. 2015;21:1-13
pubmed: 25712379
Drug Des Devel Ther. 2015 Apr 01;9:1937-53
pubmed: 25897204
Mol Genet Metab. 2016 Feb;117(2):150-6
pubmed: 26432669
Mol Genet Metab. 2016 Feb;117(2):84-94
pubmed: 26452513
Expert Opin Orphan Drugs. 2015 Nov 1;3(11):1279-1290
pubmed: 26973801
Biol Blood Marrow Transplant. 2016 Nov;22(11):2104-2108
pubmed: 27555533
Ann Thorac Surg. 2016 Oct;102(4):e329-31
pubmed: 27645974
J Inherit Metab Dis. 2017 Jan;40(1):151-158
pubmed: 27718145
Mol Genet Metab Rep. 2016 Oct 10;9:42-45
pubmed: 27761411
Mol Genet Metab. 2017 Jan - Feb;120(1-2):78-95
pubmed: 27979613
Expert Opin Orphan Drugs. 2016;4(9):941-951
pubmed: 28217429
Mol Genet Metab. 2018 Feb;123(2):112-117
pubmed: 29273385
Mol Genet Metab. 2018 May;124(1):1-10
pubmed: 29627275
Mol Genet Metab. 2018 Sep;125(1-2):18-37
pubmed: 29779902
Mol Genet Metab. 2018 Sep;125(1-2):44-52
pubmed: 29779903
EBioMedicine. 2018 Nov;37:366-373
pubmed: 30366815
Biol Blood Marrow Transplant. 2019 Jul;25(7):e226-e246
pubmed: 30772512
Mol Genet Metab Rep. 2019 Feb 05;19:100455
pubmed: 30775257
J Hum Genet. 2019 Jul;64(7):625-635
pubmed: 31019230
Clin Orthop Relat Res. 1976 Jan-Feb;(114):4-9
pubmed: 770041