Correction of lysosomal dysfunction as a therapeutic strategy for neurodegenerative diseases

Bioorganic and Medicinal Chemistry Letters

Volumn 24, Issue 14, 2014, Pages 3001-3005

  Boyd, Robert E ^a   Valenzano, Kenneth J ^a  

^a Amicus Therapeutics   (United States)

Author keywords

Gcase; Lysosomal dysfunction; Neurodegeneration

Indexed keywords

ALPHA MANNOSIDASE; ALPHA SYNUCLEIN; BETA MANNOSIDASE; CHAPERONE; GLUCOSYLCERAMIDASE; GLUCOSYLCERAMIDE; GLUCOSYLSPHINGOSINE; LYSOSOME ENZYME; MIGLUSTAT; N BUTYL 1 DEOXYNOJIRIMYCIN; SPHINGOSINE DERIVATIVE; UNCLASSIFIED DRUG; BETA GLUCOSIDASE; CENTRAL NERVOUS SYSTEM AGENTS; IMINOSUGAR 2 ACETAMIDO 1,2 DIDEOXYNORJIRIMYCIN; ISOFAGAMINE; MIGALASTAT; N ACETYL GLUCOSAMINE THIAZOLINE; PYRAZOLOPYRIMIDINE DERIVATIVE;

ALZHEIMER DISEASE; ARTICLE; BINDING AFFINITY; CELL MIGRATION; DEGENERATIVE DISEASE; ENDOPLASMIC RETICULUM ASSOCIATED DEGRADATION; ENZYME ACTIVITY; ENZYME REPLACEMENT; ENZYME SYNTHESIS; FABRY DISEASE; FLUORESCENCE SPECTROSCOPY; FRAMESHIFT MUTATION; GAIN OF FUNCTION MUTATION; GAUCHER DISEASE; GENE; GLUCOSYLCERAMIDASE 1 GENE; HUMAN; INDEL MUTATION; LOSS OF FUNCTION MUTATION; LYSOSOME STORAGE DISEASE; MISSENSE MUTATION; NUCLEAR MAGNETIC RESONANCE SPECTROSCOPY; PARKINSON DISEASE; STOP CODON; TAY SACHS DISEASE; CORRELATION ANALYSIS; DRUG TARGETING; ENZYME DEFICIENCY; GENE MUTATION; LYSOSOME; MOLECULAR PATHOLOGY; NONHUMAN; RISK FACTOR; DRUG DEVELOPMENT; DRUG EFFECTS; ENZYMOLOGY; GENETICS; METABOLISM; MUTATION; NEURODEGENERATIVE DISEASES;

DRUG DISCOVERY; GLUCOSYLCERAMIDASE; HUMANS; LYSOSOMES; MUTATION; NEURODEGENERATIVE DISEASES;

EID: 84902543452     PISSN: 0960894X     EISSN: 14643405     Source Type: Journal    
DOI: 10.1016/j.bmcl.2014.04.108     Document Type: Review

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