Page 59 - CIBERER-2015-eng

ratory approved by the Spanish Agency of Medicines and Medical Devices for the development of gene therapy protocols with hematopoietic stem cells.
The work done by our research team - in collaboration with other groups from the CIBER, including Linked Clinical Groups (i.e. H. Niño Jesús and H. Vall d’He- bron) and the Hospital Fundación Jimenez Diaz (with
whom we have formed a Joint Unit for Advanced Therapies) - is allowing us to lead global gene therapy programs on rare diseases that affect blood cells. Ad- ditionally we offer collaboration with other research- ers from the CIBER to develop new advanced thera- pies for rare diseases.
Most relevant scientific articles
cuesta-domingueZ a, leon-rico d, alVareZ l, dieZ B, StemCellReports.2015Dec8;5(6):1053-66.PubMedPMID:
Research Groups
Bodega-maYor i, Banos r, et al. BCR-JAK2 drives a my- eloproliferative neoplasm in transplanted mice. J Pathol. 2015 Jun;236(2):219-28. PubMed PMID: 25664618.
FernándeZ-garcía m, YaneZ rm, sáncheZ-domingueZ r, hernando-rodrígueZ m, Peces-BarBa m, herrera g, et al. Mesenchymal stromal cells enhance the engraftment of hematopoietic stem cells in an autologous mouse trans- plantation model. Stem Cell Res Ther. 2015;6:165. PubMed PMID: 26345192. Pubmed Central PMCID: PMC4562358.
garate Z, quintana-Bustamante o, crane am, oliVier e, Poirot l, galetto r, et al. Generation of a High Number of Healthy Erythroid Cells from Gene-Edited Pyruvate Kinase Deficiency Patient-Specific Induced Pluripotent Stem Cells.
Highlights
In March 2015, the Spanish Agency of Medicines and Medical Devices (AEMPS) has certified that the lab “CliniStem” at the CIEMAT complies with the re- quirements of Good Manufacturing Practices for the production of advanced therapy medicinal products under research. Besides, Begoña Díez presented her doctoral thesis on gene editing in hematopoietic stem cells as a potential therapeutic approach for Fanconi anemia.
In the field of Fanconi anemia, the safety of lentivi- ral vector LV-FANCA has been demonstrated and we have also carried out the validation of the FANCO- LEN gene therapy protocol, allowing the approval of the clinical protocol by the AEMPS in March 2015.
With respect to erythrocyte pyruvate kinase deficien- cy (PKLR) we have developed tools for genetic edit- ing to correct the gene defect in human hematopoie- tic progenitors. Therefore, we have also developed a partnership with an USA company to fund a clinical trial of gene therapy for deficiency PKLR, expected to be signed during the first quarter of 2016.
26549847. Pubmed Central PMCID: PMC4682065.
lóPeZ-santalla m, mancheno-corVo P, menta r, lóPeZ-Belmonte J, delarosa o, Bueren Ja, et al. Hu- man Adipose-Derived Mesenchymal Stem Cells Mod- ulate Experimental Autoimmune Arthritis by Modify- ing Early Adaptive T Cell Responses. Stem Cells. 2015 Dec;33(12):3493-503. PubMed PMID: 26205964.
molina-esteVeZ FJ, noWrouZi a, loZano ml, galY a, char- rier s, Von Kalle c, et al. Lentiviral-Mediated Gene Therapy in Fanconi Anemia-A Mice Reveals Long-Term Engraftment and Continuous Turnover of Corrected HSCs. Curr Gene Ther. 2015;15(6):550-62. PubMed PMID: 26415575.
A lentiviral vector for gene therapy of leukocyte ad- hesion deficiency type I (LAD-I) has been developed, which was part of the doctoral thesis of Diego León Rico, and obtained cum laude qualification with in- ternational mention.
Preclinical studies with mesenchymal stromal cells (MSCs) have shown that intravenous administration of MSCs significantly improve the graft of hemato- poietic stem cells in autologous transplantation models in mice, which will be applied in gene therapy protocols.
In collaboration with the biotech company Tigenix, cellular responses underlying the therapeutic effect of MSCs in a preclinical model of rheumatoid arthri- tis induced by collagen have been described. Fur- thermore, we have demonstrated that MSCs infused by intralymphatic route can modulate experimental collagen induced arthritis.
Institution: C. de I. Energéticas, Medioambientales y Tecnológicas (CIEMAT) · Contact: Avda. Complutense, 40. Edif. 70A. 28040 Madrid · Tel.: 91 346 65 18 · E.mail: [email protected] · Web: http://www.ciemat.es/
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