AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy

Molecular Therapy

Volumn 25, Issue 12, 2017, Pages 2727-2742

  Morabito, Giuseppe ^a,b   Giannelli, Serena G ^a   Ordazzo, Gabriele ^a   Bido, Simone ^a   Castoldi, Valerio ^a,c   Indrigo, Marzia ^a   Cabassi, Tommaso ^a   Cattaneo, Stefano ^a   Luoni, Mirko ^a,c   Cancellieri, Cinzia ^a   Sessa, Alessandro ^a   Bacigaluppi, Marco ^a   Taverna, Stefano ^a   Leocani, Letizia ^a,c   Lanciego, José L ^d,e   Broccoli, Vania ^a,f  

^a SAN RAFFAELE HOSPITAL   (Italy)

^b UNIVERSITY OF MILANO BICOCCA   (Italy)

^c VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

^d UNIVERSITY OF NAVARRA   (Spain)

^e CENTRO DE INVESTIGACIÓN BIOMÉDICA EN RED SOBRE ENFERMEDADES NEURODEGENERATIVAS CIBERNED   (Spain)

^f CNR   (Italy)

Author keywords

AAV; DREADD receptors; gene therapy; neurodegenerative diseases; Parkinson's disease; synucleinopathy; Tsc1; tuberous sclerosis

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ALPHA SYNUCLEIN; GLUCOSYLCERAMIDASE; BETA GLUCOSIDASE; TUBEROUS SCLEROSIS COMPLEX 1 PROTEIN; TUMOR SUPPRESSOR PROTEIN;

ADULT; ANIMAL BEHAVIOR; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BLOOD BRAIN BARRIER; CONTROLLED STUDY; DRUG EFFICACY; FOREBRAIN; GENE EXPRESSION REGULATION; GENE INACTIVATION; MOUSE; NEUROPROTECTION; NONHUMAN; SYNUCLEINOPATHY; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS PARTICLE; ADMINISTRATION AND DOSAGE; ANIMAL; BRAIN; DEPENDOPARVOVIRUS; DISEASE MODEL; ELECTROENCEPHALOGRAPHY; ENZYME ACTIVATION; GENE EXPRESSION; GENE ORDER; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HUMAN; METABOLISM; NERVE CELL; TRANSGENIC MOUSE;

ALPHA-SYNUCLEIN; ANIMALS; BETA-GLUCOSIDASE; BLOOD-BRAIN BARRIER; BRAIN; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; ELECTROENCEPHALOGRAPHY; ENZYME ACTIVATION; GENE EXPRESSION; GENE ORDER; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MICE; MICE, TRANSGENIC; NEURONS; TRANSDUCTION, GENETIC; TUMOR SUPPRESSOR PROTEINS;

EID: 85028735261     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2017.08.004     Document Type: Article

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