Update of treatment for mucopolysaccharidosis type III (sanfilippo syndrome).
Enzyme replacement therapy
Gene therapy
Hematopoietic stem cell transplantation
Mucopolysaccharidosis III
Sanfilippo syndrome
Substrate reduction therapy
Journal
European journal of pharmacology
ISSN: 1879-0712
Titre abrégé: Eur J Pharmacol
Pays: Netherlands
ID NLM: 1254354
Informations de publication
Date de publication:
05 Dec 2020
05 Dec 2020
Historique:
received:
05
07
2020
revised:
10
09
2020
accepted:
14
09
2020
pubmed:
20
9
2020
medline:
18
5
2021
entrez:
19
9
2020
Statut:
ppublish
Résumé
Mucopolysaccharidosis III (Sanfilippo syndrome, MPS III) is caused by lysosomal enzyme deficiency, which is a rare autosomal recessive hereditary disease. For now, there is no approved treatment for MPS III despite lots of efforts providing new vision of its molecular basis, as well as governments providing regulatory and economic incentives to stimulate the development of specific therapies. Those efforts and incentives attract academic institutions and industry to provide potential therapies for MPS III, including enzyme replacement therapies, substrate reduction therapies, gene and cell therapies, and so on, which were discussed in this paper.
Identifiants
pubmed: 32949598
pii: S0014-2999(20)30654-3
doi: 10.1016/j.ejphar.2020.173562
pii:
doi:
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
173562Informations de copyright
Copyright © 2020 Elsevier B.V. All rights reserved.