The Possible Risks of Gene Therapy

Understanding safety is key

Many forms of gene therapy are being researched in adults, at least initially. Some gene therapies may not work in patients with certain antibodies or other preexisting conditions. Ongoing clinical trials are being conducted across a variety of genetic conditions to determine the possible risks of treatment with gene therapy.

It’s important to know that many safety precautions are taken when developing gene therapies. Clinical trials in the United States are closely monitored by the FDA and the National Institutes of Health. Patient safety is the top priority.

Possible Risks to Be Aware Of

Gene transfer that uses AAV vectors to deliver new genetic material may have several risks:

  • As with any gene therapy, the body’s immune system could respond to the newly introduced therapeutic vector as if it were an intruder. An immune system reaction can lead to inflammation and other serious risks.
  • While the objective of using a particular vector is to direct the new gene to a specific tissue type, vectors can travel to other cells that weren’t targeted. Scientists are studying the potential safety impact of therapeutic vectors traveling to other tissue types.
  • After delivery of the gene therapy, the remaining vector particles can be released from the recipient’s body. Called vector shedding, this can occur through bodily fluids such as urine, saliva, or semen. Vector shedding raises the possibility of passing those remaining materials on to untreated individuals through close contact. The significance of vector shedding is currently being evaluated in gene therapy clinical trials.
  • Gene therapy may result in the creation of too much of the protein. The effect of this overproduction, or over-expression, could vary based on the type of protein being created. The significance of overproduction is currently being evaluated in gene therapy clinical trials.
  • For some people, gene therapy may not work at all. It is not yet clear how long the effects of gene therapy may last.

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