There looks to be significant growth in the targeting and efficiency of gene therapy based on work from researchers at Oregon Health and Science University and Stanford University, which were published today in the journal Nature.
They found that there is a protein common in the majority of cells that transports genetic material from a cell’s outer wall to the interior. This is significant because it could allow for certain treatments, those in which scientists generally use a virus to transport genetic material into cells, to do the same thing but without the potential of destroying the cell itself in the process.
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“It’s the doorway to the cell, if you like,” Michael Chapman, an OHSU biology professor and researcher who worked on the discovery, told Portland Business Journal. “The hope is to target these therapies only to diseased cells. And, having identified this protein, we can now start to understand how it interacts.”
Although gene therapy approaches have sometimes failed in the past, even causing cancer as a result, Chapman believes this discovery will first be significant for treating what he calls “low hanging fruit” conditions like inherited blindness and hemophilia B – just to start.
It will probably take years before this therapy is used for other more complicated conditions, but this is certainly a promising jumping-off point.
“What we’ve really done is removed a roadblock that’s been around for 20 years,” Chapman said. “That, in turn, should help the multitude of groups working on gene therapy make real progress. It’s really a fundamental step in the science.”
