Safe, efficient, and reproducible gene therapy of the brain in the dog models of sanfilippo and hurler syndromes

Molecular Therapy

Volumn 19, Issue 2, 2011, Pages 251-259

  Ellinwood, N Matthew ^a   Ausseil, Jérme ^b,c   Desmaris, Nathalie ^b,c   Bigou, Stéphanie ^b,c   Liu, Song ^b,c   Jens, Jackie K ^a   Snella, Elizabeth M ^a   Mohammed, Eman E A ^a   Thomson, Christopher B ^a   Raoul, Sylvie ^d   Joussemet, Béatrice ^e   Roux, Françoise ^e   Chérel, Yan ^f   Lajat, Yaouen ^d   Piraud, Monique ^g   Benchaouir, Rachid ^h   Hermening, Stephan ^h   Petry, Harald ^h   Froissart, Roseline ^g   Tardieu, Marc ⁱ   Ciron, Carine ^j   Moullier, Philippe ^j   Parkes, Jennifer ^a   Kline, Karen L ^a   Maire, Irène ^g   Vanier, Marie Thérèse ^c   Heard, Jean Michel ^b,c   Colle, Marie Anne ^f   more..

^a IOWA STATE UNIVERSITY   (United States)

^b INSTITUT PASTEUR   (France)

^c INSERM   (France)

^d NANTES UNIVERSITY HOSPITAL   (France)

^e LUNAM UNIVERSITÉ   (France)

^f CNRS   (France)

^g Centre de Biologie et de Pathologie Est   (France)

^h Amsterdam Molecular Therapeutics (AMT) BV   (Netherlands)

ⁱ BICÊTRE HOSPITAL   (France)

^j UNIVERSITÉ DE NANTES   (France)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA N ACETYLGLUCOSAMINIDASE; BACULOVIRUS VECTOR; CYCLOSPORIN; LEVO IDURONIDASE; MYCOPHENOLIC ACID; PARVOVIRUS VECTOR;

ANIMAL EXPERIMENT; ARTICLE; CONTROLLED STUDY; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; EXPERIMENTAL DOG; GENE DOSAGE; GENE TRANSFER; GENETIC TRANSDUCTION; HUNTER SYNDROME; HURLER SYNDROME; IMMUNOSUPPRESSIVE TREATMENT; NONHUMAN; REPRODUCIBILITY; SANFILIPPO SYNDROME; SEROTYPE; TREATMENT DURATION; TREATMENT RESPONSE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ADENO-ASSOCIATED VIRUS; ANIMALIA; CANIS FAMILIARIS; HEXAPODA;

EID: 79551632875     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2010.265     Document Type: Article

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