Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease)

Biochimica et Biophysica Acta - Molecular Basis of Disease

Volumn 1832, Issue 11, 2013, Pages 1906-1909

  Hawkins Salsbury, Jacqueline A ^a   Cooper, Jonathan D ^b,c   Sands, Mark S ^a  

^a WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b KING'S COLLEGE LONDON   (United Kingdom)

^c KING'S COLLEGE LONDON   (United Kingdom)

Author keywords

Batten disease; Gene therapy; Lysosomal storage disease; Neurodegeneration; Neuroinflammation; Neuronal ceroid lipofuscinosis

Indexed keywords

CASPASE 9; CYSTAFOS; LYSOSOME ENZYME; MERCAPTAMINE; MYCOPHENOLIC ACID; PALMITOYL PROTEIN THIOESTERASE; RESVERATROL;

BLOOD BRAIN BARRIER; BONE MARROW TRANSPLANTATION; DISEASE COURSE; EARLY DIAGNOSIS; ENZYME REPLACEMENT; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; MOTOR PERFORMANCE; NERVE FIBER TRANSPORT; NEURAL STEM CELL TRANSPLANTATION; NEURONAL CEROID LIPOFUSCINOSIS; NEURONAL CEROID LIPOFUSCINOSIS INFANTILE FINNISH TYPE; NONHUMAN; OXIDATIVE STRESS; PRIORITY JOURNAL; QUALITY OF LIFE; REVIEW; TIGHT JUNCTION;

BATTEN DISEASE; GENE THERAPY; LYSOSOMAL STORAGE DISEASE; NEURODEGENERATION; NEUROINFLAMMATION; NEURONAL CEROID LIPOFUSCINOSIS;

ANIMALS; DISEASE MODELS, ANIMAL; HUMANS; MEMBRANE PROTEINS; MICE; MUTATION; NEURONAL CEROID-LIPOFUSCINOSES; PHENOTYPE;

MURINAE; MUS;

EID: 84881559031     PISSN: 09254439     EISSN: 1879260X     Source Type: Journal    
DOI: 10.1016/j.bbadis.2013.05.026     Document Type: Review

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