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Most gene therapy experiments are still being conducted in animals, and those being tested in people are producing mixed results.
"Most gene therapy trials are done in people with life-threatening or terminal illnesses," Dr. Jay P. Siegel, a Food and Drug Administration official, told a Senate subcommittee.
Most gene therapy techniques use a virus to carry new genes into a cell but cannot direct the virus to insert genes at a specific site.
And before the panel learned that most gene therapy researchers were failing to comply with its guidelines to report patient side effects, Dr. Isner was singled out for failing to do so.
Viruses are used to deliver the corrective human genes in most gene therapy trials because of their natural ability to enter human cells and incorporate their genetic material into human chromosomes.
(Most gene therapy experiments consider a 1% to 2% conversion rate a success).
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Most gene therapies target a cell's DNA, using a virus to insert a corrected version of a gene, which then pumps out functional RNA and healthy protein.
Although most current gene therapy clinical trials focus on cancer and inherited diseases, multiple studies have evaluated the efficacy of gene therapy to abrogate various forms of heart disease.
Most current gene therapy clinical trials are using viral vectors that are self-replication defective.
However, most common gene therapy approaches involve the use of viral vectors, which are characterized by several safety concerns that limit their translational potential.
These concerns are discussed herein in the context of the most relevant gene therapy studies conducted so far in HIV/AIDS.
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