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Volumn 5, Issue , 2015, Pages

EXTL2 and EXTL3 inhibition with siRNAs as a promising substrate reduction therapy for Sanfilippo C syndrome

Author keywords

[No Author keywords available]

Indexed keywords

EXTL2 PROTEIN, HUMAN; EXTL3 PROTEIN, HUMAN; GLYCOSAMINOGLYCAN; HEPARAN SULFATE; MEMBRANE PROTEIN; MESSENGER RNA; N ACETYLGLUCOSAMINYLTRANSFERASE; SMALL INTERFERING RNA;

EID: 84941124110     PISSN: None     EISSN: 20452322     Source Type: Journal    
DOI: 10.1038/srep13654     Document Type: Article
Times cited : (21)

References (10)
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    • Elbashir, S.M.1
  • 6
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    • Impairment of glycosaminoglycan synthesis in mucopolysaccharidosis type IIIA cells by using siRNA: A potential therapeutic approach for Sanfilippo disease
    • Dziedzic, D., Wegrzyn, G. & Jakóbkiewicz-Banecka, J. Impairment of glycosaminoglycan synthesis in mucopolysaccharidosis type IIIA cells by using siRNA: a potential therapeutic approach for Sanfilippo disease. Eur. J. Hum. Genet. 18, 200-205 (2010).
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    • Dziedzic, D.1    Wegrzyn, G.2    Jakóbkiewicz-Banecka, J.3
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    • Gene silencing of EXTL2 and EXTL3 as a substrate deprivation therapy for heparan sulphate storing mucopolysaccharidoses
    • Kaidonis, X. et al. Gene silencing of EXTL2 and EXTL3 as a substrate deprivation therapy for heparan sulphate storing mucopolysaccharidoses. Eur. J. Hum. Genet. 18, 194-199 (2010).
    • (2010) Eur. J. Hum. Genet. , vol.18 , pp. 194-199
    • Kaidonis, X.1
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    • Molecular analysis of Sanfilippo syndrome type C in Spain: Seven novel HGSNAT mutations and characterization of the mutant alleles
    • Canals, I. et al. Molecular analysis of Sanfilippo syndrome type C in Spain: seven novel HGSNAT mutations and characterization of the mutant alleles. Clin. Genet. 80, 367-374 (2011).
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    • Improved and simple micro assay for sulfated glycosaminoglycans quantification in biological extracts and its use in skin and muscle tissue studies
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* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.