Prevalence of Anti-Adeno-Associated Virus Serotype 8 Neutralizing Antibodies and Arylsulfatase B Cross-Reactive Immunologic Material in Mucopolysaccharidosis VI Patient Candidates for a Gene Therapy Trial

Human Gene Therapy

Volumn 26, Issue 3, 2015, Pages 145-152

  Ferla, Rita ^a,b   Claudiani, Pamela ^a,b   Savarese, Marco ^a,c   Kozarsky, Karen ^d   Parini, Rossella ^e   Scarpa, Maurizio ^f,g   Donati, Maria Alice ^h   Sorge, Giovanni ⁱ   Hopwood, John J ^j   Parenti, Giancarlo ^a,b   Fecarotta, Simona ^b   Nigro, Vincenzo ^a,c   Sivri, Hatice Serap ^k   Van Der Ploeg, Ans ^l   Andria, Generoso ^b   Brunetti Pierri, Nicola ^a,b   Auricchio, Alberto ^a,b  

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

^b UNIVERSITY OF NAPLES FEDERICO II   (Italy)

^c SECOND UNIVERSITY OF NAPLES   (Italy)

^d REGENXBIO INC   (United States)

^e Neonatal Intensive Care Unit   (Italy)

^f UNIVERSITY OF PADOVA   (Italy)

^g Horst Schmidt Klinik   (Germany)

^h UNIVERSITY OF FLORENCE   (Italy)

ⁱ UNIVERSITY OF CATANIA   (Italy)

^j SOUTH AUSTRALIAN HEALTH AND MEDICAL RESEARCH INSTITUTE   (Australia)

^k HACETTEPE UNIVERSITY   (Turkey)

^l ERASMUS MEDICAL CENTER   (Netherlands)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ADENO ASSOCIATED VIRUS 8 VECTOR; CROSS REACTIVE IMMUNOLOGIC MATERIAL; IMMUNOLOGIC FACTOR; N ACETYLGALACTOSAMINE 4 SULFATASE; NEUTRALIZING ANTIBODY; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG;

ADENO ASSOCIATED VIRUS 8; ADOLESCENT; ADULT; ARSB GENE; ARTICLE; CHILD; CLINICAL ARTICLE; COHORT ANALYSIS; CONTROLLED STUDY; DRUG EFFICACY; ETHNICITY; FEMALE; FRAMESHIFT MUTATION; GENE; GENE MUTATION; GENE THERAPY; GENE TRANSFER; HUMAN; HUMAN CELL; MALE; MAROTEAUX LAMY SYNDROME; MISSENSE MUTATION; MULTICENTER STUDY; NONSENSE MUTATION; PATIENT SELECTION; PHENOTYPE; PRESCHOOL CHILD; PREVALENCE; SCHOOL CHILD; SKIN FIBROBLAST; SPLICING MUTATION; VIRAL GENE DELIVERY SYSTEM; WESTERN BLOTTING; YOUNG ADULT; BLOOD; CLINICAL TRIAL; CROSS REACTION; DEPENDOPARVOVIRUS; GENETICS; IMMUNOLOGY; ITALY; MUCOPOLYSACCHARIDOSIS VI; MUTATION; NETHERLANDS; NUCLEOTIDE SEQUENCE; PROCEDURES; STANDARDS; TURKEY;

ADENO-ASSOCIATED VIRUS;

ANTIBODIES, NEUTRALIZING; COHORT STUDIES; CROSS REACTIONS; DEPENDOVIRUS; DNA MUTATIONAL ANALYSIS; GENETIC THERAPY; HUMANS; ITALY; MUCOPOLYSACCHARIDOSIS VI; MUTATION; N-ACETYLGALACTOSAMINE-4-SULFATASE; NETHERLANDS; PATIENT SELECTION; TURKEY;

EID: 84924976964     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2014.109     Document Type: Article

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