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AAV8-Specific Immune Adsorption Column: A Treatment Option for Patients with Pre-Existing Anti-AAV8 Neutralizing Antibodies

Authors :
Kruzik, Anita
Raim, Roland
Voelkel, Dirk
Weiller, Markus
Hoellriegl, Werner
Scheiflinger, Friedrich
Rottensteiner, Hanspeter
Reipert, Birgit M
de la Rosa, Maurus
Source :
Blood; November 2019, Vol. 134 Issue: 1, Number 1 Supplement 1 p5922-5922, 1p
Publication Year :
2019

Abstract

Background: Pre-existing neutralizing antibodies (NAbs) are a major hurdle in adeno-associated virus 8 (AAV8) gene therapy and result in patients being excluded from clinical trials. Prevalence data published by Kruzik et al. suggest that up to 50% of patients have biologically relevant NAbs against AAV8, excluding them from current clinical trials. We developed a strategy to circumvent pre-existing anti-AAV8 immunity and specifically remove anti-AAV8 antibodies.

Details

Language :
English
ISSN :
00064971 and 15280020
Volume :
134
Issue :
1, Number 1 Supplement 1
Database :
Supplemental Index
Journal :
Blood
Publication Type :
Periodical
Accession number :
ejs56879380
Full Text :
https://doi.org/10.1182/blood-2019-126730