Fondazione “Leonardo Giambrone” per la Guarigione dalla Thalassemia onlus, a not-for-profit body founded in 1992 to promote the development of definitive thalassaemia therapies, divulged a press release to promote the development of gene therapy in thalassaemic patients.

Thalassaemia is a severe genetic disease represents a relevant problem for Mediterranean countries. For example, it is estimated that in Italy there are at least 7.000 thalassaemic patients.

Thanks to transfusions and new therapies reducing the toxic storage of iron (so-called chelating drugs), the life expectancy of thalassaemia patients has lengthened considerably, but still there is no treatment that allow the healing.

In the last years, the most promising therapies are bone marrow transplant (when possible) and especially the gene therapy, which modifies or replaces the mutated genes, restoring the production of normal blood.

One of the most promising research projects for thalassaemia gene therapy of involves Italian research groups (in Palermo and Cagliari) and the Memorial Sloan-Kettering Cancer Center in New York.

This project has already passed the numerous preliminary steps of the preclinical research and first in-man studies and is going to be a reality of care for Italian patients. In order to reach this point, it is necessary to provide an important structural support, concerning clinical, laboratory, but also social and family aspects. For this reason, the Foundation “Leonardo Giambrone” invites everyone to support this research project, by calling or sending an SMS to the number 45506 from June 16 to 30. More information is available on the website.