Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial

The Lancet Neurology

Volumn 16, Issue 9, 2017, Pages 712-720

  Tardieu, Marc ^a   Zérah, Michel ^b   Gougeon, Marie Lise ^c   Ausseil, Jérome ^d   de Bournonville, Stéphanie ^a   Husson, Béatrice ^a   Zafeiriou, Dimitrios ^e   Parenti, Giancarlo ^f,g   Bourget, Philippe ^b   Poirier, Béatrice ^c   Furlan, Valérie ^a   Artaud, Cécile ^c   Baugnon, Thomas ^b   Roujeau, Thomas ^h   Crystal, Ronald G ⁱ   Meyer, Christian ^j   Deiva, Kumaran ^a   Heard, Jean Michel ^c  

^a BICÊTRE HOSPITAL   (France)

^b HÔPITAL NECKER ENFANTS MALADES   (France)

^c INSTITUT PASTEUR   (France)

^d UNIVERSITÉ DE PICARDIE JULES VERNE   (France)

^e ARISTOTLE UNIVERSITY OF THESSALONIKI   (Greece)

^f UNIVERSITY OF NAPLES FEDERICO II   (Italy)

^g TELETHON INSTITUTE OF GENETICS AND MEDICINE TIGEM   (Italy)

^h Laboratoire Epsylon   (France)

ⁱ WEILL CORNELL MEDICAL COLLEGE   (United States)

^j UNIQURE   (Netherlands)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; ALPHA N ACETYLGLUCOSAMINIDASE; GLUCOSE; MYCOPHENOLATE MOFETIL; PREDNISOLONE; PROTEIN; TACROLIMUS; TUMOR NECROSIS FACTOR; ACETYLGLUCOSAMINIDASE; ALPHA-N-ACETYL-D-GLUCOSAMINIDASE; IMMUNOSUPPRESSIVE AGENT;

ADENO ASSOCIATED VIRUS 2; ADENO ASSOCIATED VIRUS 5; ANEMIA; ARTICLE; BRAIN GROWTH; CASE REPORT; CD8+ T LYMPHOCYTE; CEREBELLUM; CHILD; COGNITION; CONTROLLED STUDY; CYTOKINE PRODUCTION; DIARRHEA; DISEASE COURSE; DRUG BLOOD LEVEL; DRUG TOLERANCE; ECHOGRAPHY; EDEMA; ENZYME ACTIVITY; FEMALE; FEVER; FRANCE; GASTROENTERITIS; GENE TRANSFER; GLUCOSE BLOOD LEVEL; GREECE; HEPATOMEGALY; HUMAN; IMMUNE RESPONSE; IMMUNOSUPPRESSIVE TREATMENT; INFLAMMATION; ITALY; KIDNEY SIZE; LOSS OF APPETITE; MALE; MITRAL VALVE REGURGITATION; MUCOPOLYSACCHARIDOSIS TYPE IIIB; NECROSIS; NUCLEAR MAGNETIC RESONANCE IMAGING; PAIN; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PREOPERATIVE TREATMENT; PRESCHOOL CHILD; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; RESPIRATORY TRACT INFECTION; SANFILIPPO SYNDROME; SCAR; T LYMPHOCYTE; UNSPECIFIED SIDE EFFECT; VIRAL GENE THERAPY; VIRUS INFECTION; VIRUS SHEDDING; WHITE MATTER; ADMINISTRATION AND DOSAGE; ADVERSE EFFECTS; BRAIN; CLINICAL TRIAL; DEPENDOPARVOVIRUS; ENZYMOLOGY; GENE THERAPY; GENE VECTOR; GENETICS; INFANT; MUCOPOLYSACCHARIDOSIS III; OUTCOME ASSESSMENT; PHARMACOLOGY; PROCEDURES; SYNDROME;

ACETYLGLUCOSAMINIDASE; BRAIN; CHILD, PRESCHOOL; DEPENDOVIRUS; GENETIC THERAPY; GENETIC VECTORS; HUMANS; IMMUNOSUPPRESSIVE AGENTS; INFANT; MUCOPOLYSACCHARIDOSIS III; OUTCOME ASSESSMENT (HEALTH CARE); SYNDROME;

EID: 85023745177     PISSN: 14744422     EISSN: 14744465     Source Type: Journal    
DOI: 10.1016/S1474-4422(17)30169-2     Document Type: Article

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