Targeted Gene Therapy Offers Pain Relief

Aditi Bhargava, PhD

T1 Catalyst Award Supports Crucial Next Step in Development

By Kate Rauch

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

Aditi Bhargava, PhD, associate professor at UCSF’s 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 you’re affecting the whole house in the process — a huge negative side effect.” 

Likening her method to a Trojan horse, Bhargava’s 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.

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