The new approach is based on the protein jumping mechanism in which special molecules help to connect individual parts of the protein to a full functional structure. This allows you to “collect” large therapeutic proteins in the patient’s cells.
Technology is successfully tested on human and mouse cells. As researchers point out, it is promising for giving genes, which are considered very large for traditional genetic therapy.
Scientists have formed a prototype of the drug for the treatment of Stargardt’s disease – a common form of inherited retinal dystrophy. The studies of the method on biological security and therapeutic efficiency are currently being carried out.
Source: Ferra
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