A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

• DOI: 10.1038/mt.2011.108
• Publication Date: 2011-05-31T14:20:31Z
• Authors: Virginie Monteilhet; Samir Saheb; Sylvie Boutin; Christian Leborgne; Philippe Veron; Marie-Françoise M...; Philippe Moullier; Olivier Benveniste; Carole Masurier
• Keywords: Plasmapheresis; Adeno-associated virus

Adeno-associated viruses (AAV) are small, nonenveloped single-stranded DNA which require helper to facilitate efficient replication. These recombinant some of the most promising candidates for therapeutic gene transfer treat many genetic and acquired diseases. Nevertheless, presence humoral responses wild-type AAV common among humans is one limitations in vivo transduction efficacy using cognate vector. In this study, based on serum samples that we were able collect from various clinical situations, studied impact five plasmapheresis (PP), at 1–5 day intervals neutralizing factor (NAF) titers specific types 1, 2, 6, 8 seropositive patients with diverse pathologies immunosuppressor treatments. We show frequent sessions PP result drastic reduction NAF AAV1, undetectable levels or <1:5, mainly when initial titers, i.e., before first ≤1:20. Altogether, these results use its possible association pharmacological immunosuppressive treatments may help design optimal management therapy