A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS gene therapy in Canavan mice

Molecular Therapy

Volumn 21, Issue 12, 2013, Pages 2136-2147

  Ahmed, Seemin Seher ^a   Li, Huapeng ^a   Cao, Chunyan ^a,b   Sikoglu, Elif M ^a   Denninger, Andrew R ^c   Su, Qin ^a   Eaton, Samuel ^c   Liso Navarro, Ana A ^a   Xie, Jun ^a   Szucs, Sylvia ^d   Zhang, Hongwei ^a,f   Moore, Constance ^a   Kirschner, Daniel A ^c   Seyfried, Thomas N ^c   Flotte, Terence R ^a   Matalon, Reuben ^d   Gao, Guangping ^a,e  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^b TONGJI UNIVERSITY   (China)

^c BOSTON COLLEGE   (United States)

^d University of Texas Medical Branch School of Medicine   (United States)

^e SICHUAN UNIVERSITY   (China)

^f SOUTH DAKOTA STATE UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ASPARTOACYLASE; LIPID; MICRORNA; PARVOVIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BRAIN EDEMA; CANAVAN DISEASE; CONTROLLED STUDY; DISEASE SEVERITY; DRUG EFFICACY; GENE OVEREXPRESSION; GENE TARGETING; LETHALITY; LIPID ANALYSIS; LIPID BRAIN LEVEL; MALE; MOTOR DYSFUNCTION; MOUSE; MYELINATION; NONHUMAN; PERINATAL PERIOD; RETINOPATHY; SURVIVAL TIME; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ADENO-ASSOCIATED VIRUS; ANIMALIA; MUS; PRIMATES;

AMIDOHYDROLASES; ANIMALS; ANIMALS, NEWBORN; CANAVAN DISEASE; CENTRAL NERVOUS SYSTEM; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE KNOCKOUT TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; INJECTIONS, INTRAVENOUS; MICE; MICRORNAS; ORGAN SPECIFICITY; X-RAY DIFFRACTION;

EID: 84890120669     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.138     Document Type: Article

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