Gene silencing of EXTL2 and EXTL3 as a substrate deprivation therapy for heparan sulphate storing mucopolysaccharidoses

European Journal of Human Genetics

Volumn 18, Issue 2, 2010, Pages 194-199

  Kaidonis, Xenia ^a,b   Liaw, Wan Chin ^a   Roberts, Ainslie Derrick ^a,c   Ly, Marleesa ^a,c   Anson, Donald ^a,c   Byers, Sharon ^a,b,c  

^a SA Pathology ^*  (Australia)

^b UNIVERSITY OF ADELAIDE   (Australia)

^c UNIVERSITY OF ADELAIDE   (Australia)

Author keywords

EXTL2; EXTL3; Heparan sulphate; Mucopolysaccharidosis IIIA; Substrate deprivation therapy

Indexed keywords

EXTL2 PROTEIN; EXTL3 PROTEIN; HEPARAN SULFATE; LENTIVIRUS VECTOR; N ACETYLGLUCOSAMINYLTRANSFERASE; SHORT HAIRPIN RNA; UNCLASSIFIED DRUG;

ARTICLE; CONTROLLED STUDY; GENE EXPRESSION; GENE SILENCING; GLYCOSAMINOGLYCAN METABOLISM; HUMAN; HUMAN TISSUE; LYSOSOME; MUCOPOLYSACCHARIDOSIS; PRIORITY JOURNAL; RNA INTERFERENCE; SKIN FIBROBLAST; SUBSTRATE DEPRIVATION THERAPY; SYSTEMIC THERAPY;

CHILD; DNA PRIMERS; GENE EXPRESSION REGULATION; GENE PRODUCTS, GAG; GENE SILENCING; GENES, REPORTER; HEPARITIN SULFATE; HUMANS; LYSOSOMES; MEMBRANE PROTEINS; MUCOPOLYSACCHARIDOSES; N-ACETYLGLUCOSAMINYLTRANSFERASES; POLYMERASE CHAIN REACTION; RNA; T-LYMPHOCYTES; TRANSFECTION;

EID: 74449083518     PISSN: 10184813     EISSN: 14765438     Source Type: Journal    
DOI: 10.1038/ejhg.2009.143     Document Type: Article

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