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• p53 pathway activation by telomere attrition in X-DC primary fibroblasts occurs in 
Most relevant 
the absence of ribosome biogenesis failure and as a consequence of DNA damage. 
scientific c J, G a, M-G c, P-b l, P r. Clin Transl 
arrilloonzálezanGuánarcíaintadoernincheserona
articles
Oncol. 2013 Sep 25. [Epub ahead of print] PMID:24065372.

• Targeting Chk2 improves gastric cancer chemotherapy by impairing DNA dama- 

ge repair. Gutiérrez-González a, belda-iniesta c, barGiela-iParraGuirre J, doMinGuez G, 
García alFonso P, Perona r, sanchez-Perez i. Apoptosis. 2013 Mar;18(3):347-60. doi: 

10.1007/s10495-012-0794-2. PMID:23271172.




Highlights
GSE24.2 a peptide corresponding to an internal domain of Dyskerin (a protein 
member of the telomerase complex) has proved to induce telomerase activity by 

stabilizing hTR (The RNA component of telomerase) and increasing expression of 

TERT (The catalytic subunit of telomerase). GSE24.2 (Gestelmir) has been recent- 
ly approved by EMA for the treatment of Dyskeratosis congenita (DC). Expression 

of GSE24.2 in human fibroblast is able to protect from DNA damage detected by 
decreased H2AX foci and ATM and CHK2 phosphorylation. Due to these findings 

we have explored the use of GSE24.2 in ataxia telangiectasia human cells and 

studied the consequences in both DNA damage and ROS production. We have 
used both fibroblast and lymphoblast cell lines obtained from Corriel carrying 

different mutation in the ATM gene. Infection of A-T human cells with lentiviral 

vectors expressing GSE24.2 showed a reduction in basal levels of DNA damage, 
decreased levels of ROS, proinflammatory cytokines, and also lower levels of p38 

phosphorylation, than cells infected with control virus. Finally infection of AT cells 

with GSE24.2 is able to rescue these cells from senescence. We have also found 
that a shorter peptide than the complete GSE24.2 is also able to perfomr similar 

activities in Atcells. GSE24.2 and GSE4 loaded NPs could be delivered to AT and 
DC cells, and therefore, become an effective approach for the treatment of DC 

and other defective telomerase syndromes. As well as other diseases, harboring 

oxidative stress and inflammation.

We have cover the intellectual property of both nanoparticles and also the shorter 
version of the GSE24.2 peptide as well as the activities on oxidative stress and 

inflammation with two patents presented this year.












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