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Volumn 14, Issue 15, 2013, Pages 2050-2058

Rescue of functional CFTR channels in cystic fibrosis: A dramatic multivalent effect using iminosugar cluster-based correctors

Author keywords

CFTR correctors; Cystic fibrosis; Iminosugars; Multivalency; Protein folding diseases

Indexed keywords

CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; IMINOSUGAR; MIGLUSTAT;

EID: 84885422576     PISSN: 14394227     EISSN: 14397633     Source Type: Journal    
DOI: 10.1002/cbic.201300312     Document Type: Article
Times cited : (43)

References (47)
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    • 84885428485 scopus 로고    scopus 로고
    • Academisch Medisch Centrum), WO 2007123403 A1
    • J. M. F. G. Aerts, R. G. Boot (Academisch Medisch Centrum), WO 2007123403 A1, 2007.
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    • Aerts, J.M.F.G.1    Boot, R.G.2
  • 35
    • 37349013379 scopus 로고    scopus 로고
    • These data could reflect the lack of ER access by multivalent iminosugars 13b and 10b. However, in an unpublished study performed in our groups on Gaucher disease, these two compounds were found to be better pharmacological chaperones of Asn370Ser β-glucocerebrosidase than 5b, demonstrating their ability to access the ER (see the Supporting Information). For a review on pharmacological chaperones, see
    • These data could reflect the lack of ER access by multivalent iminosugars 13b and 10b. However, in an unpublished study performed in our groups on Gaucher disease, these two compounds were found to be better pharmacological chaperones of Asn370Ser β-glucocerebrosidase than 5b, demonstrating their ability to access the ER (see the Supporting Information). For a review on pharmacological chaperones, see J.-Q. Fan, Biol. Chem. 2008, 389, 1-11.
    • (2008) Biol. Chem. , vol.389 , pp. 1-11
    • Fan, J.-Q.1
  • 39
    • 84885419864 scopus 로고    scopus 로고
    • The fact that 1 is a strong inhibitor of ER α-glucosidase I is a major concern regarding long-term treatments, as this enzyme is involved in the elaboration of the glycan structure of glycoproteins.
    • The fact that 1 is a strong inhibitor of ER α-glucosidase I is a major concern regarding long-term treatments, as this enzyme is involved in the elaboration of the glycan structure of glycoproteins.
  • 41
    • 84885420145 scopus 로고    scopus 로고
    • For reviews concerning therapeutic approaches to protein folding diseases, see
    • For reviews concerning therapeutic approaches to protein folding diseases, see


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.