New Study Looks at Gene Therapy and Broken Bones

Researchers have found that a combination of gene therapy, stem cells, and ultrasound have healed significantly broken bones in lab animals. The technique is in its early stages, but it repaired large bone gaps during the testing period and may be able to heal badly broken bones in humans in the future.

In small breaks and fractures, the bone is able to repair itself over time and with casting. But severe breaks can leave large gaps in the bone that are not able to heal on their own. Typically, bone grafting is used to heal these breaks. This treatment involves taking bone tissue (either from elsewhere on the body or a donor) and then using it to repair the broken bone.

Dan Gazit, a senior researcher on the study and a professor of surgery at Cedars-Sinai Medical Center, indicated that there is a need for an alternative to bone grafting. Since bone grafting often involves an additional surgery, it can mean more pain and added risks for patients. Bone tissue taken from donors often does not integrate with the patient’s existing bone very well.

During the study, which was published in Science Translational Medicine, researchers implanted a collagen matrix into the bone gap and then attracted the bone’s resident stem cells. Once those cells populated, a mixture of “microbubbles” and genetic material for bone protein was injected into the site. An ultrasound wand was used to get the stem cell membranes to allow the DNA fragments in. Eventually, new bone tissue was formed.

This approach healed bone breaks in all of the lab animals that were treated. In the control group of untreated animals, the breaks did not heal.

More research—and human testing—is needed. The study was funded by private grants and the government.

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