Emptying the stores: Lysosomal diseases and therapeutic strategies

Nature Reviews Drug Discovery

Volumn 17, Issue 2, 2018, Pages 133-150

  Platt, Frances M ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

AMBROXOL; ARIMOCLOMOL; CURCUMIN; ELIGLUSTAT; GENISTEIN; LUCERASTAT; MIGALASTAT; MIGLUSTAT; PYRIMETHAMINE; VENGLUSTAT;

BIOGENESIS; BIOLOGICAL THERAPY; BLOOD BRAIN BARRIER; BONE MARROW TRANSPLANTATION; CALCIUM SIGNALING; CATABOLISM; CATALYSIS; CELL ORGANELLE; CENTRAL NERVOUS SYSTEM; ENDOPLASMIC RETICULUM; ENERGY METABOLISM; ENZYME REPLACEMENT; EXOCYTOSIS; GLYCOSYLATION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HETEROZYGOSITY; HUMAN; IMMUNOREGULATION; LIPID METABOLISM; LYSOSOME; LYSOSOME STORAGE DISEASE; MORBIDITY; MORTALITY; MULTIPLE CHRONIC CONDITIONS; NEWBORN SCREENING; NONHUMAN; PRIORITY JOURNAL; PROTEIN PROCESSING; REVIEW; ANIMAL; CLINICAL TRIAL (TOPIC); DRUG EFFECT; HOMEOSTASIS; METABOLISM; PRECLINICAL STUDY; PROCEDURES;

ANIMALS; CLINICAL TRIALS AS TOPIC; DRUG EVALUATION, PRECLINICAL; ENZYME REPLACEMENT THERAPY; HOMEOSTASIS; HUMANS; LYSOSOMAL STORAGE DISEASES; LYSOSOMES;

EID: 85041314069     PISSN: 14741776     EISSN: 14741784     Source Type: Journal    
DOI: 10.1038/nrd.2017.214     Document Type: Review

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