Gene Therapy Could Prevent Arthritis

By Lisa Raffensperger | March 13, 2013 1:22 pm

Most people who live to old age will suffer from arthritis. The conditions prevalence is growing alongside a graying world population.

However the only treatments at the moment address the symptoms rather than the causethe loss of cartilage in joints. Joint replacement is a last-ditch solution for some sufferers. Now a gene therapy approach has demonstrated promise in staving off arthritis in mice, opening the door to human testing.

The inspiration for the research came from studying children with a genetic form of arthritis that strikes early. These children are deficient in the gene for a protein called lubricin. Lubricin is thought to act as a lubricant between the bones in a joint.

Since a lack of lubricin caused arthritis, researchers thought perhaps additional lubricin could stave it off.

They tested this hypothesis by creating a strain of mice with an additional lubricin gene in their DNA. When these mice suffered an injury to their knees they didnt develop injury-induced arthritis. Inspection of the mices joints found that their cartilage resembled mice whod never been injured in the first place. Non-modified mice, on the other hand, had symptoms of arthritis just a month after injury.

Whats more, as the mice that made extra lubricin aged, their cartilage stayed youthful. That suggests the protein may protect against both common forms of arthritis: injury-related and age-related.

The treatment also works if the replacement genes are injected right into the joint itself, the researchers report in Science Translational Medicine today. Its delivery to human patients, then, could be similar to the injection of joint lubricants that some arthritis sufferers currently rely on.

However no gene therapies are currently approved by the FDA for human treatment, so this research will likely stay in the lab for some time yet.

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Gene Therapy Could Prevent Arthritis

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