Gene therapy for cross-correction of somatic organs and the CNS in mucopolysaccharidosis II in rodents and non-human primates.
AAV
Hunter syndrome
MPS II
cross-correction
gene therapy
iduronate-2-sulfatase
lysosome
non-human primates
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
08 Jun 2023
08 Jun 2023
Historique:
received:
29
07
2022
accepted:
23
03
2023
medline:
26
6
2023
pubmed:
26
6
2023
entrez:
26
6
2023
Statut:
epublish
Résumé
Mucopolysaccharidosis II (MPS II) is a rare lysosomal storage disease characterized by deficient activity of iduronate-2-sulfatase (I2S), leading to pathological accumulation of glycosaminoglycans (GAGs) in tissues. We used iduronate-2-sulfatase knockout (
Identifiants
pubmed: 37359415
doi: 10.1016/j.omtm.2023.03.014
pii: S2329-0501(23)00050-5
pmc: PMC10285185
doi:
Types de publication
Journal Article
Langues
eng
Pagination
286-302Informations de copyright
© 2023 The Authors.
Déclaration de conflit d'intérêts
N.C., R.C., K.D., B.F., E.J.G., C.L., N.L., K.P., J.S., S.T., C.T.W., and V.W.C. are employees of Takeda Development Center Americas, Inc., Cambridge, MA, USA. D.E.E., X.F., C.G., H.G., Y.H., X.L., and D.S. were employees of Takeda Development Center Americas, Inc., Cambridge, MA, USA while contributing to this study. All authors may hold stock or equity interests in Takeda Pharmaceuticals Company Limited. V.W.C. and X.L. are authors on a patent application related to this work.
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