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PubMed · 15491799

Gene therapy in the clinic: whose risks?

Richard E Ashcroft
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Abstract

Current experience with gene therapy for X-linked severe combined immunodeficiency disorder (X-SCID) suggests that we might now be at the point where it can be considered the 'standard of care'. This therapy carries an unquantified risk of leukaemia, raising important questions for regulators. How dangerous does a potentially curative therapy for a fatal illness need to be before we call it unsafe. How uncertain does a risk need to be for us to regard a therapy as still 'experimental'? Whose interests should prevail? Here I argue that in X-SCID it is the parents and children whom we should listen to first and foremost. How far does this patient-centred approach to licensing therapies extend? Can it be given a rational basis?

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Consumer Product SafetyGenetic TherapyGovernment RegulationHumansLeukemiaPractice Patterns, Physicians'Public OpinionPublic PolicyRisk AssessmentRisk FactorsSevere Combined ImmunodeficiencyUnited StatesBiomedical and Behavioral ResearchGenetics and ReproductionLegal ApproachJournal Article

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BibTeXRIS

Richard E Ashcroft. 2004. Gene therapy in the clinic: whose risks?. https://doi.org/10.1016/j.tibtech.2004.09.007

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