title: Gene therapy ethics and haemophilia: an inevitable therapeutic future?
abstract: 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 fueled 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.
authors.affiliation: Division of Hematology/Oncology, Department of Pediatrics, Weill Medical College of Cornell University, New York, NY 10021, USA. dmdimich@med.cornell.edu
authors.forename: D
authors.initials: D
authors.lastname: Dimichele
authors.forename: F G
authors.initials: FG
authors.lastname: Miller
authors.forename: J J
authors.initials: JJ
authors.lastname: Fins
mesh_terms: D026690:Ethics, Clinical; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000941:ethics*; D006467:Hemophilia A / Q000628:therapy*; D002836:Hemophilia B / Q000628:therapy*; D006801:Humans; D008297:Male; D018570:Risk Assessment