Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-
Animals
Animals, Newborn
Brain
/ drug effects
CHO Cells
Cricetulus
Disease Models, Animal
Enzyme Replacement Therapy
/ methods
Glycosaminoglycans
/ metabolism
Heparitin Sulfate
/ metabolism
Humans
Liver
/ drug effects
Lysosomal Storage Diseases
/ drug therapy
Lysosomes
/ drug effects
Mice
Mucopolysaccharidosis III
/ drug therapy
Neurons
/ drug effects
Receptor, IGF Type 2
/ metabolism
Recombinant Proteins
/ pharmacology
Sulfatases
/ pharmacology
CHO cells
DHFR
GNS
MPS IIID
enzyme replacement therapy
recombinant protein production
Journal
Molecular pharmaceutics
ISSN: 1543-8392
Titre abrégé: Mol Pharm
Pays: United States
ID NLM: 101197791
Informations de publication
Date de publication:
04 01 2021
04 01 2021
Historique:
pubmed:
16
12
2020
medline:
6
10
2021
entrez:
15
12
2020
Statut:
ppublish
Résumé
There is currently no cure or effective treatment available for mucopolysaccharidosis type IIID (MPS IIID, Sanfilippo syndrome type D), a lysosomal storage disorder (LSD) caused by the deficiency of α-
Identifiants
pubmed: 33320673
doi: 10.1021/acs.molpharmaceut.0c00831
pmc: PMC8362844
mid: NIHMS1728712
doi:
Substances chimiques
Glycosaminoglycans
0
Receptor, IGF Type 2
0
Recombinant Proteins
0
Heparitin Sulfate
9050-30-0
Sulfatases
EC 3.1.6.-
N-acetylglucosamine-6-sulfatase
EC 3.1.6.14
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Langues
eng
Sous-ensembles de citation
IM
Pagination
214-227Subventions
Organisme : NINDS NIH HHS
ID : R41 NS089061
Pays : United States
Organisme : NINDS NIH HHS
ID : U44 NS089061
Pays : United States
Organisme : NINDS NIH HHS
ID : R42 NS089061
Pays : United States
Organisme : NIGMS NIH HHS
ID : T32 GM008243
Pays : United States
Organisme : NINDS NIH HHS
ID : R01 NS088766
Pays : United States
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