The Ethics of Gene Therapy: Balancing the Risks

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The Ethics of Gene Therapy: Balancing the Risks


[Cover: discussion about how risks are balanced during risk assessment, why this is a difficult task -> proposing a set of principles and practical measures that might assist both researchers and patients, to enable more informed decisions about risk]

Ethics and gene therapy

Since its inception, gene therapy has captured the attention of the public and ethics disciplines as a therapeutic application of human genetic engineering. The latter, in particular, has lead to concerns about germline modification and questions about the distinction between therapy and enhancement. The development of the gene therapy field and its progress to the clinic has not been without controversy. Although initially considered as a promising approach for treating the genetic of disease, the field has attracted disappointment for failing to fulfil its potential. With the resolution of many of the barriers that restricted the progress of gene therapy and increasing reports of clinical success, it is now generally recognised that earlier expectations may have been premature.

High profile adverse events resulting in disproportionate media attention have prevented a greater difficulty for the field, with the death of Jesse Gelsinger in a trial of gene therapy for ornithine transcarbamylase deficiency undermining public trust of clinical research in the US. There is a danger that the gene therapy field may have become too risk-averse in response to these adverse events, and that this could manifest as fewer trials that take longer to commence. In the context of a research environment that is increasingly turning to the developing world for the expedient conduct of clinical trials, it is imper...

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...matopoietic compartment using integrating vectors particularly need to understand genotoxicity risks in relation to the risks of conventional bone marrow transplantation. A QPL could direct them to ask questions about risk, benefits and survival rates following transplantation at local centres; the prognosis of patients in the different haematopoietic gene therapy trials; the number and status of patients that developed leukaemia in the SCID-X1 gene therapy trials; and whether there are any differences between the proposed vector and the vector used in the SCID-X1 trial and any possible safety developments. This kind of guidance may help patients understand both what is known and unknown about specific applications of gene therapy.


[Could invite the GT societies to develop their own standards/guidelines and post on websites for use of their members?]

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