Gene Therapy
Study Findings from Children's Hospital of Philadelphia, Division of Hematology Broaden Understanding of Gene Therapy
Published in Gene Therapy Weekly, January 20th, 2011
2011 JAN 20 -- Research findings, 'Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction,' are discussed in a new report. "Adeno-associated viral (AAV) vectors are an extensively studied and highly used vector platform for gene therapy applications. We hypothesize that in the first clinical trial using AAV to treat hemophilia B, AAV capsid proteins were presented on the surface of transduced hepatocytes, resulting in clearance by antigen-specific CD8+ T cells and consequent loss of therapeutic transgene expression," investigators in the United States report.
"It has been previously shown that...
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Source: Gene Therapy Weekly (2011-01-20)
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