A Shocking Death from Organ Failure puts spotlight on Gene Therapy

Gene therapy is the process of correctingbody and replace a specific gene. This is the most
defective genes that cause diseases. This type ofcommonly used method of gene therapy. The
therapy is still in the early stages. No genenext option is replacing a damaged gene through
therapy products have been approved by thea process called homologous recombination. This
Food and Drug Administration (FDA) for legal sale.process involves knocking out one gene by
Gene therapy trials continue, but the success ratedisrupting its function, and then replace it with one
has not been very high, which is most of thethat works properly and has the correct genetic
reason why the FDA will not yet approve it.coding.
The first gene therapy trial took place in 1990,Abnormal genes can be repaired by selective
depending on volunteers to take parting the trial.reverse mutation. This involves selecting particular
In 1999, the therapy was thrust into the negativegenes and processing them back to their original
spotlight when a 18 year old died of organ failureform. The last option is simply to keep a gene,
just 4 days after starting treatment. His deathbut alter it to meet the specifications.
was the result of a severe immune systemEthical issues regarding gene therapy continue to
reaction to the trial drug. Trials continued untilfuel. The biggest question is what a disability is,
2003, when the FSA banned the use of retroviralwhat is normal, and who gets to decide the
vectors in blood stem cells. This decision wascriteria. Cost is another big issue. Such therapy is
made on the basis of at least 2 people in the trialvery expensive and it is debated who will pay for
developing a condition similar to leukemia. Whileit. As the technology in the area of genes
both individuals were successfully treated for it,continues, gene therapy may or may not become
trials on humans have not occurred since.a reality for those with disabilities and illnesses
There are several ways gene therapy is done. Alinked to ineffective genes.
normal gene can be inserted into a location in the