Gene therapy is a concrete application of this, in which the new way of measuring can make a world of difference. Gene therapy is mostly applied to children with a rare gene defect. Doctors bring in an harmless virus, in which scientists have packed a healthy version of the gene. Last year, for example, this form of therapy came onto the market for the muscle disease SMA under the name Zolgensma. Costs: 2.12 million dollar, the most expensive medicine in the world.
"The challenge is to properly add the gene to the virus. Methods that can measure this are crucial. If the gene is too long, it will not be taken up properly by the virus or will be sliced in pieces. If it is too short, there is a chance that the virus will take up two pieces. It is also possible that the virus particles do not have a gene built in at all. The quality of such a gene product could not be measured until now", says Heck. That will change with this new method. "Now we can measure if and which pieces the virus has absorbed. We are already being approached by several international pharmaceutical companies, who would like us to do this for them, because gene therapy is in the middle of a renaissance".
The new mass analysis can be used for a variety of large complexes too: antibodies, ribosomes, protein clots and empty or genome-packed viruses. "This really is a huge breakthrough", Heck says. "This opens up so many possibilities. I don't think we're going to realize how big this breakthrough has been until much later."
Manufacturer Thermo Fisher Scientific has announced that the new application will not be launched on the market for the time being. But the Hecklab software will become available. "We will make the developed software freely accessible", says Joost Snijder, assistant professor involved in the research. "So any lab with an Orbitrap can try it for themselves."