Helix Therapeutics LLC aims to modify disease-linked genes on certain stem cells by introducing oligonucleotides that bind to double-stranded DNA inside a cell. Ideally, the oligos will bind tightly into the groove between strands of DNA and form a triple-stranded helix, or triplex, that prevents the gene from being read. If the results of Helix's animal experiments turn out to be replicable in humans, Helix may have a way of treating, and possibly even curing, sickle-cell disease and other disorders caused by genetic mutations.
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