Therapy for Gaucher disease: Don't stop thinking about tomorrow

Molecular Genetics and Metabolism

Volumn 90, Issue 2, 2007, Pages 122-125

  Sidransky, Ellen ^a   LaMarca, Mary E ^a   Ginns, Edward I ^b  

^a NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

^b UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

Chaperone therapy; Enzyme replacement therapy; Gaucher disease; Gene therapy; Lysosomal storage diseases; Substrate reduction therapy

Indexed keywords

ADENOVIRUS VECTOR; CHAPERONE; GLUCOSYLCERAMIDASE; IMIGLUCERASE; LENTIVIRUS VECTOR; MIGLUSTAT; RETROVIRUS VECTOR;

ARTICLE; CLINICAL FEATURE; CLINICAL TRIAL; COST BENEFIT ANALYSIS; DISEASE COURSE; DRUG EFFICACY; DRUG SAFETY; ENZYME REPLACEMENT; GAUCHER DISEASE; HUMAN; IMMUNOTHERAPY; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; OUTCOME ASSESSMENT; PRIORITY JOURNAL; PROTEIN EXPRESSION; STATISTICAL SIGNIFICANCE; TREATMENT OUTCOME; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; YEAST CELL;

EID: 33846262175     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2006.09.007     Document Type: Note

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