Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

The Lancet

Volumn 388, Issue 10043, 2016, Pages 476-487

  Sessa, Maria ^a,b   Lorioli, Laura ^a,b,c   Fumagalli, Francesca ^a,b   Acquati, Serena ^a   Redaelli, Daniela ^a   Baldoli, Cristina ^b   Canale, Sabrina ^a   Lopez, Ignazio D ^b   Morena, Francesco ^d   Calabria, Andrea ^a   Fiori, Rossana ^b   Silvani, Paolo ^b   Rancoita, Paola M V ^c   Gabaldo, Michela ^a   Benedicenti, Fabrizio ^a   Antonioli, Gigliola ^b   Assanelli, Andrea ^b   Cicalese, Maria Pia ^b   del Carro, Ubaldo ^b   Sora, Maria Grazia Natali ^b   Martino, Sabata ^d   Quattrini, Angelo ^b   Montini, Eugenio ^a   Di Serio, Clelia ^c   Ciceri, Fabio ^b   Roncarolo, Maria Grazia ^a,c   Aiuti, Alessandro ^a,b,c   Naldini, Luigi ^a,c   Biffi, Alessandra ^a,b,c   more..

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b SAN RAFFAELE HOSPITAL   (Italy)

^c VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

^d UNIVERSITY OF PERUGIA   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

CEREBROSIDE SULFATASE; COMPLEMENTARY DNA; LENTIVIRUS VECTOR;

ARTICLE; CHILD; CLINICAL ARTICLE; COLONY FORMING CELL; COMPARATIVE STUDY; CONTROLLED STUDY; CYTOPENIA; DISEASE COURSE; DISEASE DURATION; DRUG EFFICACY; DRUG SAFETY; ENGRAFTMENT; FOLLOW UP; GROSS MOTOR FUNCTION MEASURE; HEMATOPOIETIC STEM CELL; HUMAN; METACHROMATIC LEUKODYSTROPHY; MUCOSA INFLAMMATION; NERVE CONDUCTION VELOCITY; NONHUMAN; OPEN STUDY; OUTCOME ASSESSMENT; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; REMYELINIZATION; STEM CELL GENE THERAPY; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; ADOLESCENT; CLINICAL TRIAL; FEMALE; GENE THERAPY; GENETICS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; INFANT; ITALY; LENTIVIRINAE; LEUKODYSTROPHY, METACHROMATIC; MALE; ONSET AGE; PRESCHOOL CHILD; PROCEDURES; TREATMENT OUTCOME;

ADOLESCENT; AGE OF ONSET; CHILD; CHILD, PRESCHOOL; FEMALE; FOLLOW-UP STUDIES; GENETIC THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFANT; ITALY; LENTIVIRUS; LEUKODYSTROPHY, METACHROMATIC; MALE; TREATMENT OUTCOME;

EID: 84988521082     PISSN: 01406736     EISSN: 1474547X     Source Type: Journal    
DOI: 10.1016/S0140-6736(16)30374-9     Document Type: Article

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