Gene therapy is feasible in stem cells from patients with Fanconi’s anemia

A team of scientific and clinical researchers from the Spanish Network of Research on Anemia of Fanconi, coordinated by the Division of Innovative Therapies of CIEMAT, demonstrates for the first time that gene therapy in stem cells of patients with Fanconi’s Anemia is feasible. Dr. Surrallés, director of the Genetics Service of Sant Pau, has participated in this finding, which has been published in the most prestigious journal of Hematology: BLOOD.

In their article, the researchers show that the gene correction procedure based on their own technology allows us to correct the pathology of the stem cells in these patients’ bone marrow.

The demonstration is based on a protocol for the genetic correction of hematopoietic stem cells in patients. After their mobilization in the patients’ blood, stem cells were corrected with clinically used viral vectors, and a very small part of them were transplanted to immunodeficient mice, in which human blood cells can be generated.

In this way, scientists have shown for the first time that through a gene therapy procedure, stem cells from patients with Fanconi’s anemia can generate disease-free human blood cells in transplanted mice.

These findings are a major boost that reinforces the researchers’ hypothesis that gene therapy will be a new way to effectively treat patients with Fanconi’s anemia. It is also of particular interest to the expectations of a clinical trial of gene therapy for these patients, which is currently underway in Spain and will soon be opened in other European countries.

This finding has been published in the most prestigious journal of Hematology: BLOOD

The news has also been highlighted by the weekly “Cell Therapy News”  on August 21 as Top Story by this newspaper.

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