Antigene therapy has raised hopes as a novel treatment strategy which employs a DNA-based drug to pinpoint light energy to a target gene.
The new finding has noted possibilities and challenges for the clinical application of this novel photo-activated DNA modulating approach.
Netanel Kolevzon and Eylon Yavin, from The Hebrew University of Jerusalem reviewed the development of triplex-forming DNA-based drugs capable of up-regulating or inhibiting gene expression in a highly targeted and selective manner.
Unlike existing antisense therapies that target RNA, an antigene drug is a triplex-forming oligonucleotide that recognizes and attaches directly to a specific DNA sequence.
By attaching a photoreactive agent to the antigene and delivering light energy to the attachment site, the light-sensitive drug complex becomes activated, triggering a cleavage or cross-linking reaction.
This photo-induced, site-specific DNA damage effectively silences the gene target.
"Many obstacles lay ahead before this approach may reach the clinic," said the authors.
However, if antigene therapy proves successful at blocking gene activity, "many diseases that are currently incurable or otherwise treatable with limited success could be potentially relevant targets for such an approach," they concluded.
The findings were published in the Oligonucleotides.