Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates

Adeno-associated virus (AAV)–based liver gene therapy has been shown to be clinically successful. However, the presence of circulating neutralizing antibodies (NABs) against AAV vector capsids remains a major challenge as it may prevent successful transduction of the target cells. Therefore, there i...

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Detalhes bibliográficos
Publicado no:Blood Adv
Main Authors: Salas, David, Kwikkers, Karin L., Zabaleta, Nerea, Bazo, Andrea, Petry, Harald, van Deventer, Sander J., Aseguinolaza, Gloria Gonzalez, Ferreira, Valerie
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2019
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6737410/
https://ncbi.nlm.nih.gov/pubmed/31501158
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2019000380
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