Bone Growth Induction in Mucopolysaccharidosis IVA Mouse.
AAV gene therapy
C-type natriuretic peptide
Morquio A syndrome
ossification
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:
08 Jun 2023
08 Jun 2023
Historique:
received:
17
04
2023
revised:
17
05
2023
accepted:
05
06
2023
medline:
29
6
2023
pubmed:
28
6
2023
entrez:
28
6
2023
Statut:
epublish
Résumé
Mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome) is caused by a deficiency of the N-acetylgalactosamine-6-sulfate-sulfatase (GALNS) enzyme, leading to the accumulation of glycosaminoglycans (GAG), keratan sulfate (KS) and chondroitin-6-sulfate (C6S), mainly in cartilage and bone. This lysosomal storage disorder (LSD) is characterized by severe systemic skeletal dysplasia. To this date, none of the treatment options for the MPS IVA patients correct bone pathology. Enzyme replacement therapy with elosulfase alpha provides a limited impact on bone growth and skeletal lesions in MPS IVA patients. To improve bone pathology, we propose a novel gene therapy with a small peptide as a growth-promoting agent for MPS IVA. A small molecule in this peptide family has been found to exert biological actions over the cardiovascular system. This work shows that an AAV vector expressing a C-type natriuretic (CNP) peptide induces bone growth in the MPS IVA mouse model. Histopathological analysis showed the induction of chondrocyte proliferation. CNP peptide also changed the pattern of GAG levels in bone and liver. These results suggest the potential for CNP peptide to be used as a treatment in MPS IVA patients.
Identifiants
pubmed: 37373036
pii: ijms24129890
doi: 10.3390/ijms24129890
pmc: PMC10298227
pii:
doi:
Substances chimiques
Keratan Sulfate
9056-36-4
Glycosaminoglycans
0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : National Institute of Health
ID : 1R01HD102545-01A1
Références
Nat Commun. 2021 Sep 9;12(1):5343
pubmed: 34504088
Drugs. 2021 Nov;81(17):2057-2062
pubmed: 34694597
Am J Hum Genet. 2012 Dec 7;91(6):1108-14
pubmed: 23200862
Endocrinology. 2007 Oct;148(10):5030-41
pubmed: 17640987
J Pharmacol Exp Ther. 2019 Sep;370(3):459-471
pubmed: 31235532
Mol Ther. 2006 Sep;14(3):316-27
pubmed: 16824801
Res Rep Endocr Disord. 2012 Dec;2012(2):65-77
pubmed: 24839594
Curr Pharm Biotechnol. 2011 Jun;12(6):931-45
pubmed: 21506915
Virology. 2004 Dec 20;330(2):375-83
pubmed: 15567432
Mol Genet Metab. 2009 Jul;97(3):196-201
pubmed: 19394256
J Biol Chem. 2003 Jul 11;278(28):25847-52
pubmed: 12736257
Mol Ther Methods Clin Dev. 2020 May 22;18:50-61
pubmed: 32577432
Curr Opin Virol. 2016 Dec;21:54-60
pubmed: 27544821
J Virol. 1999 May;73(5):3994-4003
pubmed: 10196295
Pathology. 1986 Apr;18(2):217-21
pubmed: 3093960
Clin Pharmacokinet. 2014 Dec;53(12):1137-47
pubmed: 25234648
Peptides. 2020 Oct;132:170363
pubmed: 32634451
Ital J Pediatr. 2018 Nov 16;44(Suppl 2):116
pubmed: 30442160
Mol Ther. 2010 Jun;18(6):1094-102
pubmed: 20332769
Intractable Rare Dis Res. 2020 Feb;9(1):1-9
pubmed: 32201668
J Hum Genet. 2019 Jul;64(7):625-635
pubmed: 31019230
Diagnostics (Basel). 2021 Jul 27;11(8):
pubmed: 34441285
Med Hypotheses. 2010 Dec;75(6):642-4
pubmed: 20800368
Mol Genet Metab. 2018 Sep;125(1-2):18-37
pubmed: 29779902
J Mol Histol. 2010 Oct;41(4-5):247-58
pubmed: 20721606
Mol Ther. 2008 Jul;16(7):1189-99
pubmed: 18500252
Mol Genet Metab. 2007 Jul;91(3):251-8
pubmed: 17498992
JIMD Rep. 2021 Jan 12;58(1):104-113
pubmed: 33728253
Mol Genet Metab. 2017 Jan - Feb;120(1-2):78-95
pubmed: 27979613
Br Med Bull. 2021 Sep 10;139(1):16-35
pubmed: 34453435
Drug Des Devel Ther. 2015 Apr 01;9:1937-53
pubmed: 25897204
Clin Ther. 2017 Jan;39(1):118-129.e3
pubmed: 27955919
J Virol. 1997 Sep;71(9):6823-33
pubmed: 9261407
Mol Ther. 2017 May 3;25(5):1155-1162
pubmed: 28389320
N Engl J Med. 2019 Jul 4;381(1):25-35
pubmed: 31269546
J Virol. 2003 Feb;77(4):2768-74
pubmed: 12552020
Orphanet J Rare Dis. 2011 Dec 16;6:84
pubmed: 22176730
Int J Mol Sci. 2022 May 25;23(11):
pubmed: 35682595
Osteoarthritis Cartilage. 2007 Nov;15(11):1311-7
pubmed: 17548214
J Inherit Metab Dis. 2013 Mar;36(2):235-46
pubmed: 22971960
Hum Mol Genet. 2008 Mar 15;17(6):815-24
pubmed: 18056156
Hum Mol Genet. 2005 Nov 15;14(22):3321-35
pubmed: 16219627
J Virol. 1999 Feb;73(2):1309-19
pubmed: 9882336
Biol Blood Marrow Transplant. 2019 Jul;25(7):e226-e246
pubmed: 30772512
Int J Mol Sci. 2020 Feb 23;21(4):
pubmed: 32102177
Am J Hum Genet. 2004 Jul;75(1):27-34
pubmed: 15146390
Mol Genet Metab. 2018 Sep;125(1-2):44-52
pubmed: 29779903
Clin Ther. 2015 May 1;37(5):1012-1021.e6
pubmed: 25487082
Cell. 1983 May;33(1):135-43
pubmed: 6088052
J Pediatr Orthop B. 2013 Jan;22(1):59-62
pubmed: 23010763
J Bone Miner Res. 2010 Jul;25(7):1468-86
pubmed: 20533309
Mol Genet Metab Rep. 2019 Feb 05;19:100455
pubmed: 30775257
Acta Paediatr. 2007 Apr;96(455):56-62
pubmed: 17391445
Hum Mol Genet. 2003 Dec 15;12(24):3349-58
pubmed: 14583446
Am J Physiol Endocrinol Metab. 2009 Dec;297(6):E1339-48
pubmed: 19808910
PLoS One. 2018 Dec 19;13(12):e0209517
pubmed: 30566484
J Vis Exp. 2019 Jul 18;(149):
pubmed: 31380846