Approximately one-half million Americans dice of malignant neoplastic disease yearly, doing it the 2nd prima cause of decease in the U.
The nuclei are in blue, alveolar type II cells in green and telomeres in red. Notice that lung cells treated with active telomerase present more intense telomeres indicating that they CNIO Negative results and findings in science are perhaps less newsworthy, but they are no less important.
Particularly when, as in this case, they demonstrate that a possible new therapeutic pathway against idiopathic pulmonary fibrosis and other diseases associated to short telomeres is in fact safe. Researchers from the Spanish National Cancer Research Centre CNIO have shown in a new study that the gene therapy with telomerase that they have developed, and which has proven to be effective in mice against diseases caused by excessive telomere shortening and ageing, does not cause cancer or increase the risk of developing it, even in a cancer-prone setting.
Blasco at the CNIO. Inthey designed a highly innovative strategy: These gene therapy vectors do not integrate in the genome of the host cell, thus telomerase only performs its telomere-reparative actions during a few cell divisions before the vector is diluted out.
In this manner, a potential risk associated with the activation of telomerase, such as promoting cancer, it is minimized.
But to what extent? The potential medical use of telomerase still clashes with fears surrounding a possible increased risk of cancer. The paper being published now specifically tackles this question by applying gene therapy to an animal model, a mouse, which reproduces human lung cancer and which, therefore, already has a greater risk of developing this disease.
The results are negative: That is why we chose these mice; they are animals that spontaneously develop a type of lung cancer that is very similar to the human form, which normally never appears in normal mice. How to use a double-edged sword Telomeres are at the ends of chromosomes, in the nucleus of every cell in the body; with each cell division, telomeres get a bit shorter, and when this shortening becomes excessive, the cell stops dividing and the tissue from which they are derived no longer regenerates.
We know that telomere shortening plays a key role in ageing: It has also been proven that mutations in genes related to telomeres give rise to a series of diseases called telomere syndromes, including aplastic anaemia and idiopathic pulmonary fibrosis.
Telomerase is the enzyme that repairs the telomeres, and we all have the gene that produces it. In general, telomerase is only active during embryonic development especially in the so-called pluripotent cellsbut a few days after birth, its expression is silenced.
In healthy adults, most cells do not contain telomerase: One exception are cancer cells, in which telomerase is active, and that is in fact one of the reasons why these cells divide prolifically: Telomerase has always been seen as a double-edged sword: Researchers from CNIO have witnessed the power of telomerase in action: This strategy is not applicable to humans because it would entail modifying the genome from the embryonic stage.
What is potentially applicable to humans is gene therapy, which activates telomerase where it is required, following a single local injection. Thus far, researchers have managed to demonstrate that this gene therapy with telomerase is proving to be effective in mice against infarction - contributing to the regeneration of heart tissue - aplastic anaemia, and idiopathic pulmonary fibrosis.
In all cases, gene therapy with telomerase has been seen to offer therapeutic effects. In particular, in the case of pulmonary fibrosis, which was perhaps one of the most spectacular results, telomerase was capable of halting the progression of this disease in animal models.
Although none of the mice subjected to gene therapy developed more cancer, the CNIO group wished to conduct an experiment to dispel any doubts and make it patent that safe therapy based on telomerase is possible in order to speed up the clinical application of telomerase for the treatment of diseases that currently have no cure.Jul 23, · The approval of gene therapy for leukemia, expected in the next few months, will open the door to a radically new class of cancer treatments.
Companies and universities are racing to develop these. Initial enthusiasm for gene therapy as a treatment modality was curtailed by the death of a patient participating in a dose escalation gene therapy trial in 12 While this was a trial to use gene therapy to correct a metabolic disease (ornithine transcarbamylase deficiency) and not a cancer trial, all gene therapy trials were revaluated.
Gene transfer, while a radical new type of treatment, is also the only gene therapy product to obtain regulatory approval in any global market, as demonstrated by China’s approval of Gendicine for clinical use.
76 Gendicine is a modified adenovirus that delivers the p53 gene to cancer cells and is approved for the treatment of head and. Gene Therapy Essay, Research Paper. Cancer is diagnosed in about million people in the United States yearly. Approximately one-half million Americans dice of malignant neoplastic disease yearly, doing it the 2nd prima cause of decease in the U.S.
Cystic fibrosis is an familial, fatal disease happening one time in every 2, Caucasic births and one time in every 17, African American births. Genetic Changes and Cancer. Cancer is a genetic disease—that is, cancer is caused by certain changes to genes that control the way our cells function, especially how they grow and divide..
Genes carry the instructions to make proteins, which do much of the work in our ashio-midori.comn gene changes can cause cells to evade normal growth controls and . Oct 18, · The new therapy, Yescarta, made by Kite Pharma, was approved for adults with aggressive forms of a blood cancer, non-Hodgkin’s lymphoma, who have undergone two regimens of chemotherapy that failed.