Guillermo Güenechea Amurrio

Guillermo joined the Division of Hematopoietic Innovative Therapies of CIEMAT in January 1990 and in 2004 he was appointed Head of the Unit of Safety and Risks Associated with Gene Therapy of Stem Cells, now Unit of Telomeropathies. In 1995 he defended his doctoral thesis on radioprotection conferred by ionic polysaccharides in mice and its relationship with its ability to stimulate the hematopoietic system. As a result of this work, several articles were published and a patent was filed (No. P9101063) [1, 2]. He then turned to the development of xenotransplantation models to study the effect of ex vivo expansion of human hematopoietic progenitors, as well as transduction with retroviral vectors [3-5]. In 1998 he made a 21-month stay at Hospital for Sick Children in Toronto (John Dick’s Laboratory). During this period, he carried out transduction studies of human hematopoietic progenitors with lentiviral vectors [6] and also described the existence of different types of human hematopoietic stem cells [7, 8]. In 2000, a more focused stage began to study the safety and efficacy of gene therapy with lentiviral vectors [9-15]. The researcher has also collaborated with other groups, including the collaboration with Dr. Izpisua's group (CMRB, Barcelona) for the development of lentivirally corrected Fanconi anemia hematopoietic stem cells from iPS cells [16-17 ], with the group of Dr. Ivics published 2 papers describing the impairment of iPS cells in NHEJ deficient cells and the efficient gene delivery into HSCs by transposons [18, 21], with the group of Manfred Schimdt published a work in which the safety of gene therapy of Fanconi anemia in a mouse model is described by integration site analysis of the therapeutic lentiviral vector [19]. He has recently participated in a work in which the graft and proliferative advantage of corrected progenitor cells of patients with Fanconi anemia has been demonstrated [20, 22]. More recently, we have described the targeted gene therapy into a safe harbor in human HSCs [23] and we have also generated dyskeratosis congenita-like HSCs through the stable inhibition of DKC1 [24]. He has been part of the Board of Directors of the Spanish Society of Gene and Cell Therapy as Treasurer and actively participates in the Scientific Committees of this Society and the European one. He has also been awarded a grant from a National Plan project on gene therapy and other innovative therapies for the treatment of inherited bone marrow failure syndromes (FAMOCURE). In 2019, this project has also been approved for other 3 more years (GENCURE). In relation with these projects we keep a very narrow collaboration with Dr. Rosario Perona and Leandro Sastre (IIB-CSIC, UAM, Madrid) and other national and international collaborations (Sharon Savage-NCI/NIH, USA - Felipe Prosper-CIMA, Pamplona – Zoltan Ivics-Paul Ehrlich Inst., Germany). Since 2003 he has supervised 5 Doctoral Thesis, 6 Master degree and 4 Final degree projects. He has also supervised the training of 3 technicians and actively participates in workshops, seminars and master courses. Finally, note that he is collaborator of the Agencia Estatal de Investigación (AEI), is in charge of the Biomedical Innovation biosafety and notification of genetic modified organisms, evaluates scientific articles, has participated in 37 projects, and has published 64 scientific papers between journals and book chapters, 5 of these articles as the last author.

Publications:

1. Guenechea, G., et al., Stem Cells, 1995.

2. Guenechea, G., et al., Int J Radiat Biol, 1997.

3. Guenechea, G., et al., Sangre (Barc), 1997.

4. Guenechea, G., et al., Blood, 1999.

5. Barquinero, J., et al., Blood, 2000.

6. Guenechea, G., et al., Mol Ther, 2000.

7. Dick, J.E., et al., Ann N Y Acad Sci, 2001.

8. Guenechea, G., et al., Nat Immunol, 2001.

9. Albella, B., et al., Methods Mol Biol, 2003.

10. Almarza, E., et al., Exp Hematol, 2004.

11. Laufs, S., et al., J Gene Med, 2006.

12. Almarza, E., et al., Mol Ther, 2007.

13. Gonzalez-Murillo, A., et al., Blood, 2008.

14. Guenechea, G., et al, Methods Mol Biol, 2009.

15. Gonzalez-Murillo, A., et al., Hum Gene Ther, 2010.

16. Raya, A., et al., Nature, 2009.

17. Raya, A., et al., Nat Protoc, 2010.

18. Molina-Estevez, F.J., et al., Stem Cells, 2013.

19. Molina-Estevez, F.J., et al., Curr Gene Ther, 2015.

20. Rio, P., et al, Blood 2017.

21. Holstein, M. et al, Mol Ther, 2018.

22. Rio, P. et al, Nat Med, 2019.

23. Rodriguez-Fornes, F. et al, Gene Therapy, 2020.

24. Carrascoso-Rubio, C. et al, Stem Cell Research &Therapy, 2021.