How close are we to therapies for Sanfilippo disease?

Metabolic Brain Disease

Volumn 33, Issue 1, 2018, Pages

  Gaffke, Lidia ^a   Pierzynowska, Karolina ^a   Piotrowska, Ewa ^a   Węgrzyn, Grzegorz ^a  

^a UNIVERSITY OF GDA SK   (Poland)

Author keywords

Enzyme replacement therapy; Gene therapy; Lysosomal storage; Neurodegeneration; Sanfilippo disease; Substrate reduction therapy

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ALPHA N ACETYLGLUCOSAMINIDASE; ANTIOXIDANT; EPIDERMAL GROWTH FACTOR RECEPTOR; FLAVONOID; FUSION PROTEIN; GENISTEIN; GLUCOSAMINE; GLYCOSAMINOGLYCAN; HEPARAN N SULFATASE; LENTIVIRUS VECTOR; MONOCLONAL ANTIBODY; RECOMBINANT ENZYME; SMALL INTERFERING RNA; SMALL NUCLEAR RNA; SOMATOMEDIN B; UBIDECARENONE; UNCLASSIFIED DRUG; ACETYLGLUCOSAMINIDASE; ALPHA-N-ACETYL-D-GLUCOSAMINIDASE; HEPARAN SULFATE; HEPARAN SULFATE SULFATASE; SULFATASE;

CELL THERAPY; CELL TRANSPLANTATION; ENZYME REPLACEMENT; GENE MUTATION; GENE SILENCING; GENE THERAPY; HUMAN; LYSOSOME STORAGE DISEASE; MOLECULAR BIOLOGY; MONONUCLEAR CELL; NERVE DEGENERATION; NONHUMAN; REVIEW; SANFILIPPO SYNDROME; SIGNAL TRANSDUCTION; SUBSTRATE REDUCTION THERAPY; UMBILICAL CORD; GENETICS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; MUTATION; PROCEDURES;

ACETYLGLUCOSAMINIDASE; GENETIC THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEPARITIN SULFATE; HUMANS; MUTATION; SULFATASES;

EID: 85029596768     PISSN: 08857490     EISSN: 15737365     Source Type: Journal    
DOI: 10.1007/s11011-017-0111-4     Document Type: Review

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