A new technique for precisely targeting molecules within cells may pave the way for medicines that are free of side effects, scientists say.
The same molecules do different things depending on their location, the researchers determined. By manipulating the molecules, scientists can determine exactly which locations to target, while avoiding locations that would cause harmful side effects.
"If you blocked a molecule, you blocked it regardless of what it was doing. And that usually has unwanted side effects. Almost every drug that can treat disease has side effects, either major or minor, but usually they always have something," said Zhu.
Until now, drugs have targeted molecules in a very general way. If a molecule was thought to be harmful, researchers might try to develop a drug to block it entirely.
However, Zhu's work highlights the downside of that shotgun approach. A molecule might be causing problems because of what it is doing in one part of the cell, but, at the same time, that same molecule is doing something entirely different in other parts - perhaps something tremendously important.
Shutting it down entirely would be like trying to solve the problem of traffic congestion by banning cars.
Rather than crudely trying to block a molecule regardless of its many functions, doctors can target a specific molecule doing a specific thing in a specific location.
That adds a new level of precision to the concept of precision medicine - medicine tailored exactly to a patient's needs.
The technique will also speed up the development of new treatments by letting researchers more quickly understand what molecules are doing and which should be targeted.
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