Left: Cell nucleus of a person affected by congenital dyskeratosis disease, which are harmed (green stains). Right: after the expression GSE-24-2, the cells are less harmed, as can be seen by the reduction of the green stains. Picture: CSIC
Telomeres are non-coding DNA sequences located at the endings of chromosomes. Their function is to protect the structural stability of the chromosomes.
Very often telomeres have been compared with the plastic tips that protect the endings of shoelaces. Every time that a cell and its chromosomes are divided, its telomeres get shorter. To repair the telomeres after each division is the function of an enzyme called telomerase.
In normal conditions, cells can divide about fifty or seventy times, with telomeres getting progressively shorter (and telomarase repairing them) until the cells can’t divide any more.
Experiments with animals have shown that when telomerase activity is missing, cells can’t properly divide and premature ageing appears. On the contrary, when telomerase activity is too high, cells don’t become senescent, they grow far beyond the normal limit and tumours can appear.
A group of scientists at CSIC’s Instituto de Investigaciones Biomédicas del CSIC, at the Universidad Autónoma de Madrid and Universidad Politécnica de Valencia have patented a developement that could make the obtaining of a drug possible which could protect the telomerase activity.
The patent has been distinguished proxime accessit in the madri+d best patents competition.
Small but essential
The development is a fragment of the dyskerin protein, which naturally occurs within the telomerase complex. The identified fragment, called GSE 24-2, is a small one but essential as it can restore, by itself, the telomerase enzyme function. Rosario Perona, professor at CSIC’s Instituto de Investigaciones Biomédicas “Alberto Sols”, explains that “it has been demonstrated on cultivated cells that this peptide restores the activity of telomerase in two cases of rare diseases: congenital dyskeratosis and aplastic anaemia.”
A patent licence agreement and several research contracts, including one within the program Innocash of the Spanish Ministry of Science and Innovation, have been signed with the company Advanced Medical Projects. The goal now is to develop a therapy for these two rare diseases.
Research is still at a preclinical stage, so scientists say it’s soon to speak about results. Nevertheless, the peptide could be used in other research lines focused on diseases where impaired telomerase function is implied.
Resilience to cisplatin
The group lead by Rosario Perona, in the Department of Human Diseases Experimental Models, is working on cells resilience to lung cancer treatments. A specific work line is the cellular resilience to cisplatin, used very often in chemotherapy. This drug causes the programmed cellular death of cancerous cells although, unfortunately, and like other chemotherapy drugs, it also damages healthy cells.
During experiments scientists discovered, almost accidentally, that some cells were stronger against cisplatin. “We tried to understand why and finally discovered the peptide that protects telomerase activity”.
To know more:
UTAH's page about telomeres and telomerase