title: Potential of gene therapy in bone marrow transplantation.
abstract: Gene therapy, initiated as a treatment for inherited disorders such as adenosine deaminase deficiency, is now a promising therapeutic strategy for malignancies and other acquired diseases. In particular, in the field of bone marrow transplantation (BMT) for haematological malignancies, the gene transfer of the suicide gene HSV-TK into donor lymphocytes allows control of the severe complication graft-versus-host disease (GvHD). The transfer of the HSV-TK suicide gene confers selective sensitivity to the drug ganciclovir, allowing in vivo elimination of the donor T-cells if severe GvHD occurs. In Italy, the first pilot study on delayed infusion of genetically engineered donor lymphocytes after T-depleted allogeneic BMT documented efficacy of engineered donor lymphocytes in terms of anti-tumour activity and efficiency of the suicide system. GvHD developed in 3 out of 8 patients and was successfully treated by ganciclovir administration.
authors.affiliation: Telethon Institute for Gene Therapy, Ospedale S Raffaele, Milan, Italy. marktel@tigem.it
authors.forename: S
authors.initials: S
authors.lastname: Marktel
authors.forename: C
authors.initials: C
authors.lastname: Bonini
authors.forename: C
authors.initials: C
authors.lastname: Bordignon