Gene Therapy

Research Findings from Leiden University Update Understanding of Gene Therapy

Published in Gene Therapy Weekly, April 26th, 2012

2012 APR 26 -- "For most, but not all, types of severe combined immunodeficiency (SCID) the underlying molecular defects are known, in principle allowing the cure of affected children via gene therapy. Typically such approaches have used autologous hematopoietic stem cells modified to express a therapeutic gene via gamma-retroviral vectors," scientists writing in the journal Pediatric Research report.

"Insertional mutagenesis has emerged as a significant risk for successful application of this type of gene therapy. Therefore, lentiviral vectors with a self-inactivating design have been developed. Recent advances in stem cell technology using induced...

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Source: Gene Therapy Weekly (2012-04-26)

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