作者
Eloise Hudry, Courtney Martin, Sheetal Gandhi, Bence György, Deborah I Scheffer, Dakai Mu, Steven F Merkel, Federico Mingozzi, Zachary Fitzpatrick, HJGT Dimant, Marissa Masek, Tim Ragan, Sisareuth Tan, Alain R Brisson, Servio H Ramirez, Bradley T Hyman, Casey A Maguire
发表日期
2016/4
期刊
Gene therapy
卷号
23
期号
4
页码范围
380-392
出版商
Nature Publishing Group
简介
Adeno-associated virus (AAV) vectors are showing promise in gene therapy trials and have proven to be extremely efficient biological tools in basic neuroscience research. One major limitation to their widespread use in the neuroscience laboratory is the cost, labor, skill and time-intense purification process of AAV. We have recently shown that AAV can associate with exosomes (exo-AAV) when the vector is isolated from conditioned media of producer cells, and the exo-AAV is more resistant to neutralizing anti-AAV antibodies compared with standard AAV. Here, we demonstrate that simple pelleting of exo-AAV from media via ultracentrifugation results in high-titer vector preparations capable of efficient transduction of central nervous system (CNS) cells after systemic injection in mice. We observed that exo-AAV is more efficient at gene delivery to the brain at low vector doses relative to conventional AAV, even …
引用总数
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