Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates

Human Gene Therapy Clinical Development

Volumn 26, Issue 2, 2015, Pages 113-124

  Zerah, Michel ^a,b   Piguet, Françoise ^a   Colle, Marie Anne ^c,d   Raoul, Sylvie ^e   Deschamps, Jack Yves ^c,d   Deniaud, Johan ^c   Gautier, Benoit ^a   Toulgoat, Frédérique ^f   Bieche, Ivan ^g   Laurendeau, Ingrid ^g   Sondhi, Dolan ^h   Souweidane, Mark M ^h   Cartier Lacave, Nathalie ^a   Moullier, Philippe ⁱ   Crystal, Ronald G ^h   Roujeau, Thomas ^j   Sevin, Caroline ^a,k   Aubourg, Patrick ^a,k  

^a INSERM   (France)

^b HÔPITAL NECKER ENFANTS MALADES   (France)

^c INRA UMR 703   (France)

^d LUNAM UNIVERSITÉ   (France)

^e NANTES UNIVERSITY HOSPITAL   (France)

^f CHU de Nantes   (France)

^g FACULTÉ DES SCIENCES PHARMACEUTIQUES ET BIOLOGIQUES   (France)

^h WEILL CORNELL MEDICAL COLLEGE   (United States)

ⁱ UNIVERSITÉ DE NANTES   (France)

^j Laboratoire Epsylon   (France)

^k BICÊTRE HOSPITAL   (France)

more..

Author keywords

[No Author keywords available]

Indexed keywords

CEREBROSIDE SULFATASE; PARVOVIRUS VECTOR;

ANTIBODY RESPONSE; ARTICLE; BLOOD; BRAIN MAPPING; CARTOGRAPHY; CEREBROSIDE SULFATASE GENE; CHILD; GENE; GENE THERAPY; HEMISPHERE; HUMAN; HUMAN TISSUE; LIVER; LYMPH NODE; METACHROMATIC LEUKODYSTROPHY; NERVOUS SYSTEM INFLAMMATION; NUCLEAR MAGNETIC RESONANCE IMAGING; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; SPLEEN; VIRUS GENOME; ANIMAL; BRAIN; CLINICAL TRIAL; DEPENDOPARVOVIRUS; GENE VECTOR; GENETICS; LEUKODYSTROPHY, METACHROMATIC; MACACA FASCICULARIS; MALE; METABOLISM;

MUS; PRIMATES;

ANIMALS; BRAIN; CEREBROSIDE-SULFATASE; CHILD; DEPENDOVIRUS; GENETIC THERAPY; GENETIC VECTORS; HUMANS; LEUKODYSTROPHY, METACHROMATIC; MACACA FASCICULARIS; MALE;

EID: 84964695778     PISSN: 23248637     EISSN: 23248645     Source Type: Journal    
DOI: 10.1089/humc.2014.139     Document Type: Article

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