Intrathecal administration of AAV/GALC vectors in 10–11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant

Journal of Neuroscience Research

Volumn 94, Issue 11, 2016, Pages 1138-1151

  Karumuthil Melethil, Subha ^a   Marshall, Michael S ^b   Heindel, Clifford ^a   Jakubauskas, Benas ^b   Bongarzone, Ernesto R ^b   Gray, Steven J ^a  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF ILLINOIS AT CHICAGO   (United States)

Author keywords

AAV; GALC; gene therapy; globoid cell leukodystrophy; intrathecal; Krabbe disease

Indexed keywords

BUSULFAN; GALACTOSYLCERAMIDASE; MYELIN; PARVOVIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BONE MARROW TRANSPLANTATION; BRAIN; CODON; CONTROLLED STUDY; ENZYME ACTIVITY; GLOBOID CELL LEUKODYSTROPHY; GRAFT SURVIVAL; HISTOPATHOLOGY; ISOGRAFT; LIFE EXTENSION; LIVER; MOUSE; NONHUMAN; OLIGODENDROGLIA; PRIORITY JOURNAL; SCIATIC NERVE; SEROTYPE; SPINAL CORD; TROPISM; TWITCHER MOUSE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS GENOME;

EID: 84990180427     PISSN: 03604012     EISSN: 10974547     Source Type: Journal    
DOI: 10.1002/jnr.23882     Document Type: Article

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