372. Prevalence of Anti-AAV8 Neutralizing Antibodies and ARSB Cross-Reactive Immunologic Material in MPS VI Patients Candidates for a Gene Therapy Trial
نویسندگان
چکیده
منابع مشابه
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
Recombinant vectors based on adeno-associated virus serotype 8 (AAV8) have been successfully used in the clinic and hold great promise for liver-directed gene therapy. Preexisting immunity against AAV8 or the development of antibodies against the therapeutic transgene product might negatively affect the outcomes of gene therapy. In the prospect of an AAV8-mediated, liver-directed gene therapy c...
متن کاملMutational analysis of ARSB gene in mucopolysaccharidosis type VI: identification of three novel mutations in Iranian patients
Objective(s): Mucopolysaccharidosis VI (MPS VI) or Maroteaux-Lamy syndrome is a rare metabolic disorder, resulting from the deficient activity of the lysosomal enzyme arylsulfatase B (ARSB). The enzymatic defect of ARSB leads to progressive lysosomal storage disorder and accumulation of glycosaminoglycan (GAG) dermatan sulfate (DS), which causes harmful effects on various organs and tissues an...
متن کاملAge-related prevalence of cross-reactive antibodies
B Blümel ([email protected])1,2,3,4, B Schweiger1, M Dehnert1,5, S Buda1, A Reuss1, I Czogiel1, P Kamtsiuris1, M Schlaud1, C Poethko-Müller1, M Thamm1, W Haas1 1. Robert Koch Institute, Berlin, Germany 2. Postgraduate Training for Applied Epidemiology (PAE, German FETP), Robert Koch-Institute, Berlin, Germany 3. European Programme for Intervention Epidemiology Training (EPIET), European...
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چکیده ندارد.
15 صفحه اولMaturation Pathways of Cross-Reactive HIV-1 Neutralizing Antibodies
Several human monoclonal antibodies (hmAbs) and antibody fragments, including the best characterized in terms of structure-function b12 and Fab X5, exhibit relatively potent and broad HIV-1 neutralizing activity. However, the elicitation of b12 or b12-like antibodies in vivo by vaccine immunogens based on the HIV-1 envelope glycoprotein (Env) has not been successful. B12 is highly divergent fro...
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ژورنال
عنوان ژورنال: Molecular Therapy
سال: 2015
ISSN: 1525-0016
DOI: 10.1016/s1525-0016(16)33981-8