Gene Therapy for Hemophiliacs Promising

BOSTON, Ma-Scientists successfully used a form of gene therapy to help reduce spontaneous bleeding in hemophiliacs. Preliminary findings reported several patients were treated with weakened viruses for the inherited bleeding disease.

This practice was thrust into the media after a Tucson man died in an unrelated gene therapy experiment last year. Scientists have been trying to use gene therapy for the past 20 years, however these are the first positive results from a trial for hemophilia A.

More than 13,500 Americans suffer from the genetic condition that causes abnormal bleeding.

Gene therapy is considered a new branch of medicine. Normal genes are copied and replaced to compensate for malfunctioning genes in a patient.

The patients in the hemophilia experiment had marble-sized clumps of gene-corrected cells inserted into their abdomens. Each clump contained 100-400 million cells each. The six patients, who ranged in age from 20 to 72, experienced different results. Four volunteers were able to decrease the amount of medication they were taking. Two of the patients had fewer episodes of spontaneous bleeding before the therapy.

Doctors say the treatment will need to be improved to achieve higher levels of the clotting factors but this is the first sign of therapeutic benefits for hemophiliacs.

Information from the Boston Globe.

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