Therapeutic approaches for lysosomal storage diseases: A patent update

Recent Patents on CNS Drug Discovery

Volumn 8, Issue 2, 2013, Pages 91-109

  Urbanelli, Lorena ^a   Sagini, Krizia ^a   Polidoro, Mario ^a   Brozzi, Alessandro ^a   Magini, Alessandro ^a,b   Emiliani, Carla ^a  

^a UNIVERSITY OF PERUGIA   (Italy)

^b UNIVERSITY OF UDINE   (Italy)

Author keywords

Autophagic lysosomal system; Autophagy; Cell based therapy; Cholesterol removal; Endosomal lysosomal dysfunction; Enhanced exocytosis therapy; Enzyme biodistribution; Enzyme replacement therapy; Enzyme targeting; Gene therapy; Lysosomal storage disorders; Neurodegenerative diseases; Pharmacological chaperones; Proteostasis regulation; Substrate reduction therapy

Indexed keywords

ENZYME ACTIVATOR;

ANIMAL; ENZYME REPLACEMENT; GENE THERAPY; HUMAN; LYSOSOME STORAGE DISEASE; METHODOLOGY; PATENT; REVIEW;

ANIMALS; ENZYME ACTIVATORS; ENZYME REPLACEMENT THERAPY; GENETIC THERAPY; HUMANS; LYSOSOMAL STORAGE DISEASES; PATENTS AS TOPIC;

EID: 84885661755     PISSN: 15748898     EISSN: 22123954     Source Type: Journal    
DOI: 10.2174/15748898113089990002     Document Type: Article

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