Finger Therapy: Zinc Manipulators
Two decades ago, gene therapy was declared a revolutionary method in medicine, but the development of this direction is very slow, there are few achievements, and several patients have already died in clinical trials. One of the main reasons for this failure is the difficulty in controlling the incorporation of the desired therapeutic genes into the patient's chromosomes. When using viruses for this, genes are inserted into the chromosomes of infected cells in an arbitrary place, and extremely undesirable consequences are possible, up to and including violation of the structure and functioning of existing genes.
But scientists working under the guidance of 1982 Aaron Klug, Nobel Laureate in Chemistry, have developed a method that allows therapeutic genes to be inserted into the patient’s chromosomes with surgical precision using modified “zinc fingers”. So-called zinc-containing proteins that bind to DNA and in a living cell participate in the regulation of reading the genetic code - in particular, their work provides different activity of certain genes in different types of cells. “Zinc fingers” bind only to specific nucleotide sequences, which allows manipulating genes with surgical accuracy.
The authors developed synthetic versions of these proteins called zinc-fingered nucleases. They recognize specific specific DNA sequences, allowing manipulation of specific genes without affecting neighboring ones. The new method is already undergoing clinical trials involving 100 young patients with diabetes. At the next stage of work, it is planned to test the method for the treatment of patients with spinal cord injuries.
The experimental results also indicate that the new method is suitable for blocking genes that cause the development of various diseases. This makes it a fundamental technique not only for medical, but also for animal research.
We also recall other successes in gene therapy - curing erectile dysfunction using a modified herpes virus (“For Healthy Sex”) or transplanting human vision genes into mice (“Visibility is excellent”).
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