AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

In a clinical trial for adeno-associated virus serotype 1 (AAV-1)–mediated gene transfer to muscle for lipoprotein lipase (LPL) deficiency, 1 subject from the high-dose cohort experienced a transient increase in the muscle enzyme creatine phosphokinase (CPK) 4 weeks after gene transfer. Simultaneous...

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Bibliographic Details
Main Authors: Mingozzi, Federico, Meulenberg, Janneke J., Hui, Daniel J., Basner-Tschakarjan, Etiena, Hasbrouck, Nicole C., Edmonson, Shyrie A., Hutnick, Natalie A., Betts, Michael R., Kastelein, John J., Stroes, Erik S., High, Katherine A.
Format: Artigo
Language:Inglês
Published: American Society of Hematology 2009
Subjects:
Gene Therapy
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC2744569/
https://ncbi.nlm.nih.gov/pubmed/19506302
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2008-07-167510
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