An American study has allowed us to develop a method to prevent the effects on DNA caused by this rare genetic form of anemia.
The Division of Hematology at the University of Washington School of Medicine in Seattle, a group of researchers has improved gene therapy against Fanconi anemia. This disease is very rare at present in Italy affecting about 170 people and the only hope of cure is bone marrow transplant. The procedure tries to reduce the time of transduction, lowering the oxygen tension in the cell growth with the introduction of some reducing agents that counteract the effects of free radicals.
Thanks to these special conditions has been an increase in cell growth and increased cell survival.
In 2002 in Naples, thanks to genetics services of an ASL was possible to make the first transplant using stem cells from umbilical cord of a healthy baby, who donated their cells for one of two sisters suffering from Fanconi’s anemia.
The manager of the Department of Genetics of ASL Naples 1 Rita Calzone said “The result is very exciting, since we use the most appropriate therapies to the sensitivity of patients’ cells, allowing transplants from donors compatible relatives from unrelated donors or partially compatible, have increased the chances of success for bone marrow transplants in patients with Fanconi anemia.” The results of the research are published in Gene Therapy.
