Medication from the aging stem cells successfully tested on mice

Small molecules to restore the telomerase and lengthening telomeres in the culture of stem cells of patients with hereditary telomeric disease. Substances added to the water of mice that transplanted stem cells with the same mutations. As a result, in these cells telomerase began to work normally, but telomeres lengthened. Article published in the journal Cell Stem Cell.

DNA polymerase (the enzymes that replicate DNA) are not able to copy the ends of molecules, and each cell division the chromosomes become a little shorter. So without losing important information at the ends of chromosomes are telomeres — non-coding stretches of DNA. They have their protective function, telomeres do not give different chromosomes to stick together among themselves.

Telomere length associated with cellular senescence when they become too short, the cell triggers a mechanism of aging and death, to avoid gluing chromosomes and damage to genetic material. Stem cells divide a lot (because it is their primary function), and they have the enzyme telomerasethat lengthens telomeres and allows the cells to be immortal.

Mutations that disrupt the telomeres, causing a number of hereditary diseases. One of the mechanisms of these diseases is mutation in the gene of ribonuclease, which regulates the formation of telomerase RNA template, which enzyme builds telomeres. If off the damaged gene of ribonuclease (PAPD5), formed normal telomerase RNA and telomeres lengthened.

Scientists from the United States under the leadership of Sunita Agarwal (Suneet Agarwal) from the Boston children’s hospital found a small molecule BCH001, which specifically inhibited ribonuclease mutant. The effect of the substance tested for the culture of stem cells of patients with congenital dyskeratosis, hereditary telomeric disease.

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