Innovative treatments for lysosomal diseases

Best Practice and Research: Clinical Endocrinology and Metabolism

Volumn 29, Issue 2, 2015, Pages 275-311

  Cox, Timothy M ^a  

^a UNIVERSITY OF CAMBRIDGE   (United Kingdom)

Author keywords

chaperone; gene transfer; inhibitors of biosynthesis; innovation; lysosome; organelle; protein targeting; sphingolipids; substrate reduction; therapeutic

Indexed keywords

AFEGOSTAT; AMBROXOL; CHAPERONE; ELIGLUSTAT; GENISTEIN; GLYCOPROTEIN; IMINOSUGAR; MIGLUSTAT; PARVOVIRUS VECTOR;

AUTOPHAGY; CELL FUNCTION; CELL ORGANELLE; CELL TRANSFER; CLINICAL TRIAL (TOPIC); CONTROLLED CLINICAL TRIAL (TOPIC); EXOCYTOSIS; EXPERIMENTAL THERAPY; FABRY DISEASE; GAUCHER DISEASE; GENE TRANSFER; GLYCOGEN STORAGE DISEASE TYPE 2; GM2 GANGLIOSIDOSIS; HEAT SHOCK RESPONSE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; LYSOSOME; LYSOSOME STORAGE DISEASE; MACROPHAGE; MOLECULAR PATHOLOGY; NEUROPATHOLOGY; NIEMANN PICK DISEASE; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); REVIEW; VIRAL GENE THERAPY; GENE THERAPY; LYSOSOMAL STORAGE DISEASES; MOLECULARLY TARGETED THERAPY;

GENETIC THERAPY; HUMANS; LYSOSOMAL STORAGE DISEASES; MOLECULAR TARGETED THERAPY; THERAPIES, INVESTIGATIONAL;

EID: 84929507122     PISSN: 1521690X     EISSN: 15321908     Source Type: Journal    
DOI: 10.1016/j.beem.2015.01.001     Document Type: Review

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