Gene therapy for lysosomal storage diseases: Progress, challenges and future prospects

Current Pharmaceutical Design

Volumn 17, Issue 24, 2011, Pages 2558-2574

  Seregin, Sergey S ^a   Amalfitano, Andrea ^a,b  

^a Michigan State University ^*  (United States)

^b MICHIGAN STATE UNIVERSITY   (United States)

Author keywords

Adaptive immune responses; Enzyme replacement therapy; Gene therapy; Innate immune responses; Lysosomal storage disease

Indexed keywords

AGALSIDASE ALFA; AGALSIDASE BETA; CYCLOSPORIN A; DEXAMETHASONE; FEBRAZYME; GALSUFASE; GALSULFASE; GENDICINE; IDURONATE 2 SULFATASE; IMIGLUCERASE; IMMUNOGLOBULIN; LARONIDASE; LENTIVIRUS VECTOR; METHOTREXATE; MIGLUSTAT; PARVOVIRUS VECTOR; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; RETROVIRUS VECTOR; RITUXIMAB; UNCLASSIFIED DRUG;

ADENO ASSOCIATED VIRUS; ADENOVIRUS; AUTOSOMAL RECESSIVE INHERITANCE; CARDIOMYOPATHY; COGNITIVE DEFECT; DISEASE SEVERITY; ENZYME THERAPY; FABRY DISEASE; GAUCHER DISEASE; GENE THERAPY; GENE TRANSFER; GLYCOGEN STORAGE DISEASE TYPE 2; HEAD AND NECK CANCER; HEART DISEASE; HEPATOSPLENOMEGALY; HUMAN; HYPERTENSION; IMMUNE DEFICIENCY; KIDNEY FAILURE; LYSOSOME STORAGE DISEASE; MUCOPOLYSACCHARIDOSIS; NEUROLOGICAL COMPLICATION; NIEMANN PICK DISEASE; NONHUMAN; PRIORITY JOURNAL; REVIEW; SEIZURE; STEM CELL TRANSPLANTATION; STROKE;

ADAPTIVE IMMUNITY; ANIMALS; DISEASE MODELS, ANIMAL; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; IMMUNITY, INNATE; LYSOSOMAL STORAGE DISEASES; MUTATION;

EID: 80053496078     PISSN: 13816128     EISSN: 18734286     Source Type: Journal    
DOI: 10.2174/138161211797247578     Document Type: Review

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