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CSIC BIO090- Compounds that induce telomerase activity and its application on dyskeratosis congenita, premature aging syndromes and other pathologies
A Spanish Research Institute patented new compounds that induce telomerase activity, useful for the treatment of diseases with telomerase activity alterations and cellular senescence, such as premature aging syndromes, dyskeratosis congenita, neurodegenerative diseases, cancer and other pathologies.
We look for companies interested in the license of the patent and the development of a treatment
Specification sheet:
DESCRIPTION
The expression of a dyskerin fragment, the genetic supressor element (GSE) termed GSE 24-2, induces telomerase activity. The induction could also be obtained by the administration of the correspondent 55 aminoacids-long peptide. These compounds are useful for the treatment of diseases that alter telomerase activity or with cellular senescence, such as premature aging syndromes, Werner syndrome, dyskeratosis congenita, aplastic anemia and could be possibly applied to vascular aging, cancer, neurodegenerative diseases, Fanconi anemia, atherosclerosis and Bloom syndrome.
The peptide expression reactivates telomerase activity in cell lines derived from dyskeratosis congenita patients and in telomerase-deficient human cell lines. Moreover, the expression of GSE24-2 is able to rescue dyskeratosis congenita derived fibroblasts from premature senescence. These cells are able to grow continuously up to 140 days. On the other hand, the administration of the peptide rescues telomerase activity by a direct action over telomeres Compounds could be useful for tissue regeneration treatments of epithelial or hematopoietic cells or even for immortalization of eukaryotic cells for research use or biotechnology processes
INNOVATIVE ASPECTS
This reagent is a peptide or cDNA that can be delivered to cells that harbor telomerase activity defects. These defects make the cell either to senesce and die or alternatively to become tumoral. The cells that are mainly affected are those of the immune system such as is the case in Dyskeratosis congenita and the patients usually suffer and dye from bone marrow failure and death is caused mainly by infections. It is a novel reagent since there is no alternative therapy for these patients
COMPETITIVE ADVANTAGES
To develop a drug for rare diseases such as dyskeratis congenita, Werner syndrome or aplastic anemia. The drug could be approved as an orphan drug. On the other hand the size of the peptide is small and that would facilitate delivery to patients
KEYWORDS
Dyskerin, dyskeratosis congenita, cellular senescence, telomerase, bone marrow failure, GSE24-2
PATENT
P200502511 applied for 2005-10-14
PCT/ES06/070152 applied for 2006-10-11
P200703106 applied for 2007-11-23
CONTACT
José Manuel González
email: jm.gonzalez@orgc.csic.es
phone: +34 91 585 49 56
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