Gene therapy for paediatric leukaemia

Raphaël F. Rousseau, Catherine M. Bollard, Helen E. Heslop

Research output: Contribution to journalArticlepeer-review

6 Scopus citations


Improvements in the chemotherapeutic and transplant regimens have had a significant impact in improving survival rates for paediatric leukaemia. However, there are still important problems to address including what options are available for patients with chemoresistant disease and what strategies are available to avoid the concerns regarding the toxicity associated with highly cytotoxic treatment regimens. Gene therapy and immunotherapy protocols hold great promise. Using gene transfer of a marker gene, a number of biological issues in the therapy of leukaemia have been addressed. For example, by gene marking autologous bone marrow grafts it has been possible to demonstrate that infused marrow contributes to relapse in acute and chronic myeloid leukaemias. In the allogeneic transplant setting, genetically modified T-cells have proven valuable for the prophylaxis and treatment of viral diseases and may have an important role in preventing or treating disease relapse. Gene transfer is also being used to modify tumour function, enhance immunogenicity, and confer drug-resistance to normal haematopoietic stem cells. With the continued scientific advancements in this field, gene therapy will almost certainly have a major impact on the treatment of paediatric leukaemia in the future. 2001

Original languageEnglish (US)
Pages (from-to)663-674
Number of pages12
JournalExpert Opinion on Biological Therapy
Issue number4
StatePublished - 2001


  • Adoptive immunotherapy
  • Gene dierapy
  • Leukaemia
  • Paediatric leukaemia
  • Vaccines

ASJC Scopus subject areas

  • Pharmacology
  • Biochemistry, Genetics and Molecular Biology(all)
  • Genetics
  • Immunology


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