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Use of telomerase reverse transcriptase for treatment of Dyskeratosis Congenita
EP3848056
The CNIO has developed a novel method to treat dyskeratosis congenita (DKC). The inventors have found a novel method to treat dyskeratosis congenita based on the use of a nucleic acid vector comprising a coding sequence for telomerase reverse transcriptase (TERT).
The vectors of the invention have a therapeutic benefit in dyskeratosis congenital as can be seen in the experiments performed in a mouse model of the disease. Non-integrative nucleic acid vectors are preferred, for example, adeno-associated virus (AAV). Using non-integrative viral vectors seems to be particularly advantageous. For example, non-integrative vectors do not cause any permanent genetic modification; the vectors target to adult tissues, avoiding having the subjects under the effect of constitutive telomerase expression from early stages of development, and additionally, non-integrative vectors