American scientists have developed a fundamentally new method of gene therapy, which makes it possible to effectively integrate the necessary genes into the human genome and control their activity. According to experts, this development can finally bring the gene therapy methods closer to widespread application in clinical practice.

In order to be able to control the activity of new genes (and thus protect the patient from possible side effects, such as the development of cancer or leukemia), scientists from the Children's Hospital in Boston proposed to integrate into the "therapeutic" gene, introduced into the human genome, a specific A fragment of DNA - the so-called ribozyme. Ribozyme has the ability to stop the synthesis of proteins encoded by the genes adjacent to it, but its activity can be controlled with the help of medications, which makes it possible to use it as a "switch" for genes introduced into the genome.

"One of the factors hampering the widespread use of gene therapy is the relatively high incidence of side effects associated with the modification of the human genome," said Dr. Richard Mulligan, one of the authors of the study, in an interview with "BBC". - For example, it is known that children who have undergone gene therapy are more likely to develop leukemia and other malignant tumors, which makes one think about the prospect of such a "therapy". But I hope that we managed to at least partially solve this problem.

Medicus. En.

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