Potential of gene therapy in bone marrow transplantation

S. Marktel, C. Bonini, C. Bordignon

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Pages (from-to)1-6
Number of pages6
Issue number1
Publication statusPublished - 1999

ASJC Scopus subject areas

  • Immunology and Allergy
  • Pharmacology (medical)
  • Pharmacology, Toxicology and Pharmaceutics(all)


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