Targeted Gene Therapy Offers Relief from Pain and Inflammation

Posted: May 11, 2012 at 1:11 pm

By Kate Rauch on May 11, 2012

Chronic pain affects an estimated 116 million Americans and costs $635 billion each year in medical treatment and lost productivity.

Aditi Bhargava, PhD

Aditi Bhargava, PhD, associate professor at UCSFs School of Medicine, is using a technique known as RNA interference (RNAi) to develop a gene therapy system that sends specific commands to certain neurons, or nerve cells, telling them to turn off pain, or stop inflammation.

The current treatments for pain dull everything, Bhargava said. You have a little fire in the kitchen, but your only solution is a fire hose that floods the entire house. You put out the fire, but youre affecting the whole house in the process a huge negative side effect.

Likening her method to a Trojan horse, Bhargavas novel therapeutic approach essentially hides the pain-silencing commands, carried by distinct proteins that affect cellular function, inside other proteins which bind only to the troublemaker cells. Once attached, they release their hidden power.

We want to target the small or medium neurons that sense pain, while leaving other neurons unaffected, she said. Were hoping that while you reduce pain, for example, youll still be able to chew or not drool.

The data from her proof-of-concept animal studies look promising, Bhargava said. I believe that this project has tremendous translational potential to turn what we learn into concrete benefits for patients.

Targeted pain and inflammation relief could also be used to treat illnesses, such as Inflammatory Bowel Disease (IBD), an autoimmune condition. This approach has potential to not only minimize unwanted side effects, but save costs, as it sends very small amounts of drug therapies to the targeted cells.

Targeted delivery of drug would reduce the dose required to treat, reduce cost, and most importantly, reduce unwanted side effects.

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Targeted Gene Therapy Offers Relief from Pain and Inflammation

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