Gene therapy ethics and haemophilia: An inevitable therapeutic future?

Donna Dimichele, F. G. Miller, J. J. Fins

Research output: Contribution to journalReview articlepeer-review

21 Scopus citations


Haemophilia was recognized early on as an ideal candidate for a gene transfer approach to therapy. In the past decade, gene transfer experimentation in the haemophilias has indeed played an integral role in furthering the science in the global field of gene therapy. However, these expectations have placed haemophilia gene transfer researchers under pressure to succeed in a scientific domain in which successes are infrequent and progress is necessarily slow. These same expectations have also fuelled the perception of gene therapy as the inevitable therapeutic goal for the youngest children with haemophilia. In this paper, we will discuss the ethical implications of this perception in light of anticipated benefits, acceptable risk, perceived consumer need and the unknown cost of this intervention. A framework for the future study and therapeutic implementation of gene transfer technology in this specific population is proposed. Public debate on this issue that includes the voices of the intended beneficiaries, especially the parents of the youngest children with haemophilia and the children themselves, is encouraged.

Original languageEnglish (US)
Pages (from-to)145-152
Number of pages8
Issue number2
StatePublished - Mar 2003


  • Gene theraphy
  • Haemophilia
  • Haemophilic treatment
  • Medical ethics

ASJC Scopus subject areas

  • Hematology


Dive into the research topics of 'Gene therapy ethics and haemophilia: An inevitable therapeutic future?'. Together they form a unique fingerprint.

Cite this