Antisense Technology

Researchers at University Medical Center, Department of Human Genetics have published new data on antisense technology

Published in Gene Therapy Weekly, January 6th, 2011

2011 JAN 6 -- A new study, 'Progress in therapeutic antisense applications for neuromuscular disorders,' is now available. "Neuromuscular disorders are a frequent cause of chronic disability in man. They often result from mutations in single genes and are thus, in principle, well suited for gene therapy," investigators in Leiden, Netherlands report.

"However, the tissues involved (muscle and the central nervous system) are post-mitotic, which poses a challenge for most viral vectors. In some cases, alternative approaches may use small molecules, for example, antisense oligonucleotides (AONs). These do not deliver a new gene, but rather modulate existing...

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Source: Gene Therapy Weekly (2011-01-06)

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