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31/08/2017

Study with the collaboration of VHIR demonstrates that gene therapy is feasible in stem cells of patients with Fanconi’s anaemia

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31/08/2017

Through a gene therapy procedure, stem cells of patients with Fanconi's anaemia can generate disease-free human blood cells transplanted in mice.

A team of scientific and clinical researchers from the Spanish Research Network on Fanconi's anaemia, coordinated by http://bit.ly/2vwujan" CIEMAT's Innovative Therapy Division, shows for the first time that gene therapy in stem cells of patients with Fanconi's Anaemia is feasible The following are part of this team: doctors Cristina Díaz de Heredia and Raquel Hladun, of the Group of http://bit.ly/2iMPRt5 Translational Research in Child and Adolescent Cancer of the Vall d'Hebron Research Institute and members of the http://bit.ly/2eK5jSg" Oncology and Child Haematology Department of Vall d'Hebron, of which Dr. Díaz de Heredia is the clinical head. This finding has been published in the most prestigious journal on Haematology "http://bit.ly/2glnhus Bood . The researchers show that the genetic correction procedure based on their own technology allows the pathology of bone marrow stem cells to be corrected for these patients.The demonstration is based on a protocol for the gene correction of haematopoietic stem cells of the patients. After its mobilization in the blood of the patients, the stem cells were corrected with viral vectors of clinical use, and a very small part of the same was transplanted to immunodeficient mice, in which it is possible the generation of human blood cells.In this way, scientists have shown for the first time that through a gene therapy procedure, stem cells of patients with Fanconi's anaemia can generate disease-free human blood cells transplanted in mice.These discoveries represent a great boost that reinforces the hypothesis of researchers according to which gene therapy will become a new way of effectively treating patients with Fanconi's anaemia. Likewise, it is of interest to the expectations of a clinical trial of gene therapy of these patients, which is currently ongoing in Spain, and will soon be opened to other European countries. The news has also been highlighted by the weekly http://bit.ly/2gsisDm Cell Therapy News as Top Story.

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