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Gene Therapy Improves Vision In Nearly Blind Patients.
Posted on:
28 April 2008, source: ScienceDaily
In a clinical trial at The Children's Hospital of Philadelphia, researchers from The University of Pennsylvania have used gene therapy to safely restore vision in three young adults with a rare form of congenital blindness. Although the patients have not achieved normal eyesight, the preliminary results set the stage for further studies of an innovative treatment for this and possibly other retinal diseases.
An international team led by The University of Pennsylvania, The Children's Hospital of Philadelphia, the Second University of Naples and the Telethon Institute of Genetics and Medicine (both in Italy), and several other American institutions reported their findings in an online article in the New England Journal of Medicine.
>> Read full story
Boy develops leukemia after gene therapy in UK.
Posted on:
18 December 2007, source: Reuters
A three-year-old "bubble boy" undergoing pioneering gene therapy in London has developed leukemia, marking another setback for the experimental treatment.
Doctors at Great Ormond Street Hospital said on Tuesday the boy had been successfully treated for SCID-X1, or x-linked severe combined immunodeficiency, often known as "baby in the bubble syndrome", but had developed leukemia two years later.
The news is a blow to the treatment program at the London hospital, which has a worldwide reputation from treating sick children.
>> Read full story ; see also X-SCID statement
FDA lifts ban on trial after investigating death.
Posted on:
27 November 2007, source: Nature News
A clinical trial of gene therapy in which a woman died this summer is due to recommence, thanks to a decision from the US Food and Drug Administration (FDA) announced yesterday. The experimental therapy for rheumatoid arthritis was stopped after one of the participants, 36-year-old Jolee Mohr, died in July. Autopsy findings presented in September showed that Mohr died of a massive fungal infection complicated by major internal bleeding, and that the gene therapy itself may not have been to blame (see Gene therapy might not have caused patient's death). Independently of the gene therapy, Mohr was taking several drugs for her arthritis that suppress the immune system, which is a risk factor for this type of infection.
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FDA Statement on Gene Therapy Clinical Trial.
Posted on:
26 July 2007, source: FDA
On July 24, 2007 the U.S. Food and Drug Administration (FDA) was informed by Targeted Genetics Corporation of Seattle about the death of a patient who received an investigational gene therapy product in a clinical trial for the treatment of active inflammatory arthritis.
FDA's condolences go to the patient's family.
FDA is providing this preliminary information in recognition of the public's interest in these types of new therapies.
Targeted Genetics notified FDA earlier that a patient in its trial experienced a serious adverse event. Even though the cause of the illness wasn't known, and is still uncertain, the agency immediately placed the trial on clinical hold--meaning no further product can be administered and no new patients can be enrolled.
The product that was being studied uses a particle called a vector that is designed to deliver treatment genes to target cells.
>> Read full story
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