Gene therapy is in danger of being overhyped, expert says

doi:10.1136/bmj.39566.632859.DB

BMJ 2008;336:977 (3 May), doi:10.1136/bmj.39566.632859.DB

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Gene therapy is in danger of being overhyped, expert says

Geoff Watts

¹ London

The first 150 words of the full text of this article appear below.

Two reports published online this week by the New England Journalof Medicine have described the use of gene therapy to correctthe causes of one form of congenital blindness. Although thegains in both cases were modest, media reports have greetedthem with jubilation and the suggestion that the results canbe extrapolated to a wide range of eye conditions.

This reaction has prompted one researcher who recognises thevalue of the new work to regret that, once again, enthusiasmfor gene therapy may be running dangerously ahead of its actualachievements.

Infants born with Leber’s congenital amaurosis have profoundvisual impairment throughout childhood and become completelyblind by early middle age. Previous work has shown that in patientswith this condition seven genes normally active in the cellsof the retinal pigment epithelium (RPE) show mutations.

The two research groups—one based at the University ofPennsylvania’s Scheie Eye . . . [Full text of this article]

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