News Column

New Gene Therapy Study Findings Recently Were Reported by Researchers at CIEMAT (Targeted gene therapy and cell reprogramming in Fanconi anemia)

August 28, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Data detailed on Biotechnology have been presented. According to news originating from Madrid, Spain, by NewsRx correspondents, research stated, "Gene targeting is progressively becoming a realistic therapeutic alternative in clinics. It is unknown, however, whether this technology will be suitable for the treatment of DNA repair deficiency syndromes such as Fanconi anemia (FA), with defects in homology-directed DNA repair."

Our news journalists obtained a quote from the research from CIEMAT, "In this study, we used zinc finger nucleases and integrase-defective lentiviral vectors to demonstrate for the first time that FANCA can be efficiently and specifically targeted into the AAVS1 safe harbor locus in fibroblasts from FA-A patients. Strikingly, up to 40% of FA fibroblasts showed gene targeting 42 days after gene editing. Given the low number of hematopoietic precursors in the bone marrow of FA patients, gene-edited FA fibroblasts were then reprogrammed and re-differentiated toward the hematopoietic lineage. Analyses of gene-edited FA-iPSCs confirmed the specific integration of FANCA in the AAVS1 locus in all tested clones. Moreover, the hematopoietic differentiation of these iPSCs efficiently generated disease-free hematopoietic progenitors."

According to the news editors, the research concluded: "Taken together, our results demonstrate for the first time the feasibility of correcting the phenotype of a DNA repair deficiency syndrome using gene-targeting and cell reprogramming strategies."

For more information on this research see: Targeted gene therapy and cell reprogramming in Fanconi anemia. Embo Molecular Medicine, 2014;6(6):835-48. (Wiley-Blackwell -; Embo Molecular Medicine -

The news correspondents report that additional information may be obtained from P. Rio, Division of Hematopoietic Innovative Therapies, CIEMAT, CIBERER, Madrid, Spain Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz (IIS-FJD, UAM), Madrid, Spain. Additional authors for this research include R. Banos, A. Lombardo, O. Quintana-Bustamante, L. Alvarez, Z. Garate, P. Genovese, E. Almarza, A. Valeri, B. Diez, S. Navarro, Y. Torres, J.P. Trujillo, R. Murillas, J.C. Segovia, E. Samper, J. Surralles and Gregory (see also Biotechnology).

Keywords for this news article include: Biotechnology, Spain, Madrid, Europe, Fibroblasts, Hypoplastic, Gene Therapy, Hematopoietic, Bioengineering, Fanconi Anemia, Congenital Anemia, Metabolic Diseases, Bone Marrow Diseases, Hematologic Diseases, Connective Tissue Cells, Hemic and Lymphatic Diseases, DNA Repair Deficiency Disorders.

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Source: Gene Therapy Weekly

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