Gene Therapy to Offer Effective Treatment for Osteoporosis

Gene Therapy to offer Effective Treatment for Osteoporosis

Researchers have developed an alternative to bone grafts that could help alleviate the long-term hospitalization, disability, and considerable costs to the health system associated with non-healing fractures.

Maxim Bez and colleagues developed a two-step gene therapy method coupled with FDA-approved ultrasound and microbubbles that completely healed the nonunion fractures in pigs within eight weeks of treatment. Study was published in American Association for the Advancement of Science on May 2017. In the U.S., around 100,000 people suffer from fractures, which fail to heal properly, resulting in nonunion fractures, and more than 2 million bone grafts are performed around the world annually in attempts to treat these challenging injuries. However, harvesting fresh bone from patients is painful and donated grafts from tissue banks frequently fail to integrate due to graft rejection.

Team of researchers placed a collagen scaffold at the site of the fracture to provide a support for developing bone progenitor cells. They injected microbubbles mixed with genetic material for a bone growth factor. Pulses of sound from an ultrasound wand promoted uptake of the growth factor DNA by progenitor cells, which stimulated bone growth. Ultrasound and microbubbles do not trigger inflammation and expression of the introduced gene was undetectable after 10 days. Other gene therapies rely on viral vectors to deliver their cargo, which is risky as viruses can permanently integrate into the genome and later promote cancer or set off lethal immune responses

According to Osteoporosis Treatment Market report published by Coherent Market Insights, osteoporosis treatment involves preventing and treating fractures and using medication to strengthen bones. Treatment usually includes medications such as Bisphosphonates are the most common drugs prescribed for the treatment of osteoporosis. However, these treatments are not very effective. Gene therapy could offer potential treatment for osteoporosis as this technique was proven to be minimally invasive and promoted total bone healing with comparable strength to gold-standard graft procedures. This system has potential to be used in different tissue engineering applications and treatment of osteoporosis.

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