Generation and characterization of an immunodeficient mouse model of mucopolysaccharidosis type II.

Humans Animals Mice Mucopolysaccharidosis II / therapy Mice, Inbred NOD Mice, SCID Iduronate Sulfatase / genetics Glycosaminoglycans
CRISPR/Cas9 Glycosaminoglycans Hunter syndrome Iduronate-2-sulfatase Mucopolysaccharidosis type II NSG

Journal

Molecular genetics and metabolism
ISSN: 1096-7206
Titre abrégé: Mol Genet Metab
Pays: United States
ID NLM: 9805456

Informations de publication

Date de publication:
04 2023
Historique:
received: 04 01 2023
revised: 06 02 2023
accepted: 07 02 2023
medline: 25 4 2023
pubmed: 7 4 2023
entrez: 6 4 2023
Statut: ppublish

Résumé

Mucopolysaccharidosis type II (Hunter syndrome, MPS II) is an inherited X-linked recessive disease caused by deficiency of iduronate-2-sulfatase (IDS), resulting in the accumulation of the glycosaminoglycans (GAG) heparan and dermatan sulfates. Mouse models of MPS II have been used in several reports to study disease pathology and to conduct preclinical studies for current and next generation therapies. Here, we report the generation and characterization of an immunodeficient mouse model of MPS II, where CRISPR/Cas9 was employed to knock out a portion of the murine IDS gene on the NOD/SCID/Il2rγ (NSG) immunodeficient background. IDS

Identifiants

DOI: 10.1016/j.ymgme.2023.107539 PMID: 37023503 PMC: PMC10705040
pubmed: 37023503
pii: S1096-7192(23)00169-5
doi: 10.1016/j.ymgme.2023.107539
pmc: PMC10705040
mid: NIHMS1947048
pii:
doi:

Substances chimiques

Iduronate Sulfatase EC 3.1.6.13
Glycosaminoglycans 0

Types de publication

Journal Article Research Support, N.I.H., Extramural

Langues

eng

Sous-ensembles de citation

IM

Pagination

107539

Subventions

Organisme : NINDS NIH HHS
ID : P30 NS062158
Pays : United States
Organisme : NIAID NIH HHS
ID : R21 AI163731
Pays : United States
Organisme : NIGMS NIH HHS
ID : T32 GM113846
Pays : United States

Informations de copyright

Copyright © 2023. Published by Elsevier Inc.

Références

Annu Rev Biochem. 2010;79:181-211
pubmed: 20192759
Eur J Pediatr. 2008 Mar;167(3):267-77
pubmed: 18038146
J Inherit Metab Dis. 2001 Nov;24(6):675-80
pubmed: 11768586
Hum Gene Ther. 2017 Aug;28(8):626-638
pubmed: 28478695
Mol Ther. 2017 May 3;25(5):1155-1162
pubmed: 28389320
Mol Ther. 2018 Apr 4;26(4):1127-1136
pubmed: 29580682
Int J Mol Sci. 2021 May 23;22(11):
pubmed: 34070997
J Gene Med. 2020 Sep;22(9):e3205
pubmed: 32335981
Stem Cell Reports. 2015 May 12;4(5):835-46
pubmed: 25866157
Hum Mol Genet. 2006 Apr 1;15(7):1225-36
pubmed: 16505002
Hum Gene Ther. 2023 Jan;34(1-2):8-18
pubmed: 36541357
Hum Gene Ther. 1996 Mar 1;7(4):537-49
pubmed: 8800749
Cell Stem Cell. 2009 Sep 4;5(3):310-9
pubmed: 19733542
EMBO Mol Med. 2018 Jul;10(7):
pubmed: 29884617
Clin Chem. 1997 Aug;43(8 Pt 1):1325-35
pubmed: 9267309
JCI Insight. 2016 Jun 16;1(9):e86696
pubmed: 27699273
Clin Genet. 2020 Jan;97(1):89-102
pubmed: 31231794
Dev Reprod. 2019 Jun;23(2):79-92
pubmed: 31321348
Methods Mol Biol. 2019;1916:105-111
pubmed: 30535688
Hum Gene Ther. 2022 Dec;33(23-24):1279-1292
pubmed: 36226412
J Immunol. 2005 May 15;174(10):6477-89
pubmed: 15879151
J Inherit Metab Dis. 2007 Nov;30(6):924-34
pubmed: 17876721
Acta Paediatr Suppl. 2002;91(439):98-9
pubmed: 12572850
Mol Ther. 2004 Jun;9(6):856-65
pubmed: 15194052
Nat Biotechnol. 2018 Sep;36(8):765-771
pubmed: 30010673
Genet Med. 2006 Aug;8(8):465-73
pubmed: 16912578
Mol Ther. 2021 May 5;29(5):1853-1861
pubmed: 33508431
Mol Genet Metab. 2021 May;133(1):8-34
pubmed: 33741271
Am J Transplant. 2016 Feb;16(2):389-97
pubmed: 26588186
EMBO Mol Med. 2022 Apr 7;14(4):e14297
pubmed: 35298086
Mol Ther Methods Clin Dev. 2015 Feb 11;2:14068
pubmed: 26052536
Gene Ther. 2023 Apr;30(3-4):288-296
pubmed: 35835952
Mol Ther Methods Clin Dev. 2020 Sep 20;19:261-274
pubmed: 33102618
J Comp Physiol Psychol. 1979 Feb;93(1):74-104
pubmed: 221551
Immun Inflamm Dis. 2016 Aug 28;4(4):427-440
pubmed: 27980777
Stem Cells Transl Med. 2017 Feb;6(2):352-368
pubmed: 28191778
Mol Genet Metab. 2020 Aug;130(4):262-273
pubmed: 32631737
Pediatrics. 2011 May;127(5):e1258-65
pubmed: 21518713
Blood. 2003 Mar 1;101(5):2054-63
pubmed: 12406886
PLoS One. 2017 Feb 16;12(2):e0172435
pubmed: 28207863
Mol Genet Metab Rep. 2023 Jan 20;34:100956
pubmed: 36704405
Gene Ther. 2004 Nov;11(21):1590-8
pubmed: 15356673
Nat Commun. 2017 May 31;8:15464
pubmed: 28561021
Hum Gene Ther. 1996 Feb 10;7(3):283-90
pubmed: 8835216
Hum Gene Ther. 2015 Jun;26(6):357-66
pubmed: 25761450
Mol Ther. 2022 Dec 7;30(12):3587-3600
pubmed: 36299240
Sci Rep. 2016 Mar 31;6:23579
pubmed: 27030542
Int J Mol Sci. 2021 Jul 23;22(15):
pubmed: 34360653

Auteurs

Miles C Smith (MC)

Department of Genetics, Cell Biology and Development, University Minnesota, Minneapolis, MN, United States; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States.

Lalitha R Belur (LR)

Department of Genetics, Cell Biology and Development, University Minnesota, Minneapolis, MN, United States; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States.

Andrea D Karlen (AD)

Department of Genetics, Cell Biology and Development, University Minnesota, Minneapolis, MN, United States; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States.

Kelly Podetz-Pedersen (K)

Department of Genetics, Cell Biology and Development, University Minnesota, Minneapolis, MN, United States; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States.

Olivia Erlanson (O)

Department of Genetics, Cell Biology and Development, University Minnesota, Minneapolis, MN, United States; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States.

Kanut Laoharawee (K)

Department of Genetics, Cell Biology and Development, University Minnesota, Minneapolis, MN, United States; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States.

Justin Furcich (J)

Department of Pediatrics, University of Minnesota, Minneapolis, MN, United States.

Troy C Lund (TC)

Department of Pediatrics, University of Minnesota, Minneapolis, MN, United States.

Yun You (Y)

Mouse Genetics Laboratory, University of Minnesota, Minneapolis, MN, United States.

Davis Seelig (D)

Comparative Pathology Shared Resource, University of Minnesota, St. Paul, MN, United States.

Beau R Webber (BR)

Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States; Department of Pediatrics, University of Minnesota, Minneapolis, MN, United States; Stem Cell Institute, University of Minnesota, Minneapolis, MN, United States; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, United States.

R Scott McIvor (RS)

Department of Genetics, Cell Biology and Development, University Minnesota, Minneapolis, MN, United States; Center for Genome Engineering, University of Minnesota, Minneapolis, MN, United States; Stem Cell Institute, University of Minnesota, Minneapolis, MN, United States; Masonic Cancer Center, University of Minnesota, Minneapolis, MN, United States. Electronic address: mcivo001@umn.edu.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female

Evaluation of Low-Value Services Across Major Medicare Advantage Insurers and Traditional Medicare.

Ciara Duggan, Adam L Beckman, Ishani Ganguli et al.
1.00
Humans United States Aged Cross-Sectional Studies Medicare Part C

Effectiveness of Virtual Yoga for Chronic Low Back Pain: A Randomized Clinical Trial.

Hallie Tankha, Devyn Gaskins, Amanda Shallcross et al.
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Generation and characterization of an...
questionsmedicales.fr › Eucaryotes › Animaux › Generation and characterization of an...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Rodentia › Muridae › Murinae › Souris › Generation and characterization of an...
questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Erreurs innées du métabolisme › Maladies lysosomiales › Mucopolysaccharidoses › Mucopolysaccharidose de type II › Generation and characterization of an...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Rodentia › Muridae › Murinae › Souris › Lignées consanguines de souris › Souris de lignée NOD › Generation and characterization of an...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Rodentia › Muridae › Murinae › Souris › Souches mutantes de souris › Souris SCID › Generation and characterization of an...
questionsmedicales.fr › Enzymes et coenzymes › Enzymes › Hydrolases › Esterases › Sulfuric ester hydrolases › Iduronate 2-sulfatase › Generation and characterization of an...
questionsmedicales.fr › Glucides › Polyosides › Glycosaminoglycanes › Generation and characterization of an...