Marta Perez Rodriguez, Paula Rio and Juan Antonio Burin.
The pre -clinical study, in which Spanish researchers participated for the first time in mouse models, showed the effectiveness of genetic therapy in the live body (directly managed) to Fenonnic anemia. This work, published in the magazine natureOpen the door to change the procedures for correcting blood diseases of a genetic nature: one of the previous treatments that has already been shown in patients with this rare disease In the treatments of the live bodyA much simpler system that would allow the expansion of the application of treatment to more patients.
At work, led San Rafael Telelithon Institute for genetic therapy de MilanoParticipated in a Spanish team belonging to the Biomedical Innovation Unit at the Center for Research, Environmental and Technology (CIEMAT), which is the field of rare diseases at the Biomedical Medical Research Center in the network (It will justifyThe Institute of Health Research Jiminiz Diaz Foundation (IIS-FJD).
Fanconi anemia is a complex disease that is often manifested in the age of children It affects the bone marrow stem cells Subordinate Sick. It is characterized by the gradual hatching of blood cells, which is frequently translated into severe inflammation Bone marrow failure.
In a clinical study published in the magazine Lancet In December 2024, Spanish researchers first showed the effectiveness of previous genetic therapy to correct bone marrow failure for patients with fansci anemia. This treatment is Collect and purify the sick stem cellsFollowed by correcting its genetic defect, and finally reintegrating the corrected stem cells into patients.
Treatment is much simpler
In this new experimental work, the strategy used is completely different. In this case, the therapeutic Lentivial transmission was given directly by Endovenous Road to newborn mice That suffered from this disease. As a result of this simple treatment, they noticed that a small percentage of the sister of the bone marrow patient showed the correction of the hereditary defect. From that moment on, the corrected cells have been gradually expanded in the bone marrow and animal blood. next to, Avoid corrected cells failed bone marrow When the animals were treated with a drug that harms pathological cells, which reflects the ability of this strategy to prevent bone marrow failure in this disease model.
Before treating mice with Vanconi paragraphs, Italian team researchers showed the possibility Blood -forming stem cells Among the mice made of healthy births, they have also obtained moderate therapeutic benefits in other diseases models, such as ADA deficiency (severe form of severe immune deficiency associated with the absence of lymphocytes in the blood) and bone bone bone (affected disease). “The fact that the corrected stem cells in Vanconi paragraphs show a multiplication feature that made this disease an ideal model for poor treatment in viruses in the live body. Thanks to our experience in the disease, we start in cooperation with researchers at the Tiget Institute in Milan,” explains “ Juan Burin, of the Ciemat, Cibeer and IIS-FJD team Participant in the study.
“The results obtained in this pioneering work open new views to treat Blood diseasesIn particular of Fanconi anemia, through much simpler systems that are currently used, which would allow treatment to be applied to more patients. Paula RioFrom the same research team.
This study Opening new views of the genetic treatment of diseases, Which you will undoubtedly need supplementary works to confirm the various experimental models, the safety associated with the management of Lentiviraal, capable of combining therapeutic genes into Cell genome.
In short, the work shows a very simplified way for rExplain the genetic therapy for mono -originally diseases of the blood cells.
The information published in the medical writing contains data, data and data from official institutions and health professionals. However, given any questions related to your health, consult the opposite health specialist.
