Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice

Gene Therapy

Volumn 20, Issue 9, 2013, Pages 913-921

  Heldermon, C D ^a   Qin, E Y ^b   Ohlemiller, K K ^c   Herzog, E D ^b   Brown, J R ^d   Vogler, C ^e   Hou, W ^f   Orrock, J L ^g   Crawford, B E ^d   Sands, M S ^b  

^a UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^b WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c WASHINGTON UNIVERSITY   (United States)

^d Zacharon Pharmaceuticals Inc   (United States)

^e SAINT LOUIS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^f UNIVERSITY OF FLORIDA   (United States)

^g UNIVERSITY OF WISCONSIN MADISON   (United States)

Author keywords

behavioral; MPS IIIB; Sanfilippo

Indexed keywords

ACETYLGLUCOSAMINIDASE; BETA GLUCURONIDASE; GLYCOSAMINOGLYCAN; LENTIVIRUS VECTOR; PARVOVIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; AUDITORY THRESHOLD; CIRCADIAN RHYTHM; COMPARATIVE STUDY; CONTROLLED STUDY; ENZYME ACTIVITY; EVOKED BRAIN STEM AUDITORY RESPONSE; GENE THERAPY; HEARING; LIFESPAN; MOTOR PERFORMANCE; MOUSE; NONHUMAN; PRIORITY JOURNAL; SANFILIPPO SYNDROME; TREATMENT RESPONSE; VIRUS PARTICLE;

ADENO-ASSOCIATED VIRUS; ANIMALIA; MUS;

ACETYLGLUCOSAMINIDASE; ANIMALS; ANIMALS, NEWBORN; BRAIN; CIRCADIAN RHYTHM; DEPENDOVIRUS; GENETIC THERAPY; GENETIC VECTORS; HUMANS; LENTIVIRUS; LIVER; LUNG; MALE; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; MOTOR ACTIVITY; MUCOPOLYSACCHARIDOSIS III; MYOCARDIUM; TREATMENT OUTCOME;

EID: 84883881326     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2013.14     Document Type: Article

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