Gene therapy helps improve sight of the blind

Tue, 29 Apr 2008 06:59:59 -0400

Two new human trials of a gene therapy for inherited blindness have demonstrated that injecting replacement genes under the retinas of the blind can spur at least partial sight in some volunteers. In one trial in the UK, one of three blind volunteers was able to navigate a maze after being treated. In another, the sight of all three volunteers taking part in a U.S. trial was improved somewhat. In the UK trial, researchers delivered replacement RPE65 genes using a harmless virus--a common tactic in gene therapy. And scientists feel that they may be close to offering the therapy to the blind around the world.

"This really paves the way for developing a treatment for people who have so far had no prospect of a cure," Robin Ali, a UCL ophthalmologist at UCL, told the Guardian. "If everything goes well, it's possible that within two to three years it might be approved for use in the clinic."

- read the article in the Guardian

ALSO: That success helped propel a big rise in the shares of Targeted Genetics, but not big enough to get over the $1-per-share mark. Report

Biotech co. defends gene therapy trial standards

Tue, 07 Aug 2007 06:59:59 -0400

The death of Jolee Mohr in a gene therapy trial undertaken by Seattle-based Targeted Genetics has once again thrown a spotlight on the safety record of the whole gene therapy field. The family wants to know why she was recruited for a study that offered her next to no chance of a medical benefit. The 15-page consent agreement is also being scrutinized, as the company insists that it did nothing improper in pursuing testing of tgAAC94, which uses a specially engineered virus to transport a gene. When working properly, the therapy produces proteins that eliminate the inflammatory molecules that cause arthritis. But now questions are being raised about breaches in the consent process as the family finds out that the consent agreement was signed on first sight--rather than studied--and that the principal investigator suggested she volunteer rather than having someone else make the proposal. It all represents another huge setback for gene therapy, which has been periodically roiled over the years by the unexpected deaths of trial participants.

- here's the report from The Washington Post

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