Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain

Human Gene Therapy

Volumn 28, Issue 6, 2017, Pages 510-522

  Golebiowski, DIane ^a   Van Der Bom, Imramsjah M J ^a   Kwon, Churl Su ^b   Miller, Andrew D ^c,h   Petrosky, Keiko ^c   Bradbury, Allison M ^d   Maitland, Stacy ^a   Kühn, Anna Luisa ^a   Bishop, Nina ^a   Curran, Elizabeth ^c   Silva, Nilsa ^c   GuhaSarkar, Dwijit ^a   Westmoreland, Susan V ^c   Martin, Douglas R ^d   Gounis, Matthew J ^a   Asaad, Wael F ^e,f,g   Sena Esteves, Miguel ^a  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^b MASSACHUSETTS GENERAL HOSPITAL   (United States)

^c HARVARD MEDICAL SCHOOL   (United States)

^d AUBURN UNIVERSITY   (United States)

^e Brown University Program in Neurosurgery   (United States)

^f BROWN UNIVERSITY   (United States)

^g RHODE ISLAND HOSPITAL   (United States)

^h Animal Health Diagnostic Center   (United States)

Author keywords

AAV; adeno associated virus; gene therapy; hexosaminidase; intracranial delivery; Tay Sachs disease

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; BETA N ACETYLHEXOSAMINIDASE ALPHA CHAIN; BETA N ACETYLHEXOSAMINIDASE BETA CHAIN; ENDOTOXIN; NEUTRALIZING ANTIBODY; BETA N ACETYLHEXOSAMINIDASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY BLOOD LEVEL; ANTIBODY TITER; APATHY; ARTICLE; ATAXIA; B LYMPHOCYTE; BRAIN NECROSIS; BRAIN WEIGHT; CHOREA; CONTROLLED STUDY; DRUG DOSE COMPARISON; DRUG SAFETY; DYSKINESIA; ENZYME ACTIVITY; FEMALE; FOOD INTAKE; GRAY MATTER; HISTOPATHOLOGY; LATERAL BRAIN VENTRICLE; LIMB WEAKNESS; MACACA FASCICULARIS; MALE; NEUROLOGIC DISEASE; NEUROTOXICITY; NONHUMAN; T LYMPHOCYTE; THALAMUS; VIRAL GENE DELIVERY SYSTEM; VOLUNTARY MOVEMENT; WHITE MATTER; ANIMAL; CHEMISTRY; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE EXPRESSION; GENE THERAPY; GENE VECTOR; GENETICS; GM2 GANGLIOSIDOSIS; INTRACEREBROVENTRICULAR DRUG ADMINISTRATION; METABOLISM; NECROSIS; NERVE CELL; PATHOLOGY; PROCEDURES; PROTEIN SUBUNIT; TRANSGENE;

ANIMALS; APATHY; BETA-N-ACETYLHEXOSAMINIDASES; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DYSKINESIAS; FEMALE; GANGLIOSIDOSES, GM2; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; GRAY MATTER; INJECTIONS, INTRAVENTRICULAR; MACACA FASCICULARIS; MALE; NECROSIS; NEURONS; PROTEIN SUBUNITS; THALAMUS; TRANSGENES; WHITE MATTER;

EID: 85020747917     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2016.109     Document Type: Article

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