Molecular Therapies

Research from H. Zayed and colleagues has provided new data on molecular therapies

Published in Gene Therapy Weekly, September 6th, 2007

According to a study from the United States, "Spliceosome-mediated RNA trans-splicing (SMaRT) is an emerging technology for the repair of defective pre-messenger RNA (pre-mRNA) molecules. It is especially useful in the treatment of genetic disorders involving large genes."

"Although viral vectors have been used for achieving long-lasting expression of trans-splicing molecules, the immunogenicity and suboptimal safety profiles associated with viral-based components could limit the widespread application of SMaRT in the repair of genetic defects. Here, we tested whether the non-viral Sleeping Beauty (SB) transposon system could mediate stable delivery of trans-splicing...

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Source: Gene Therapy Weekly (2007-09-06)

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