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Precision Medicine News

UVA Gene Mutation Research Method Speeds Precision Medicine

A new approach to gene editing may make it quicker and cheaper to perform precision medicine research.

Gene editing research for precision medicine

Source: Thinkstock

By Jennifer Bresnick

- A team from the University of Virginia School of Medicine has developed a quicker way to examine the impact of gene mutation on patient health, potentially changing the way cancer labs conduct research into precision medicine and personalized therapies.

The methodology, which uses a virus similar to HIV to replace normal genes with specific mutations, may even be speedier and more cost effective than the CRISPR gene editing technology that currently forms the basis for much of the industry’s cutting-edge genomics work.

"Every patient shouldn't receive the same treatment. No way. Not even if they have the same syndrome, the same disease," said UVA researcher J. Julius Zhu, PhD, who led the team that created the new technique. "It's very individual in the patient, and they have to be treated in different ways."

The process of understanding and testing a specific mutation’s impact on disease development and the usefulness of particular therapies has thus far been slow and painful, said Zhu, who holds positions in UVA's Department of Pharmacology and the UVA Cancer Center.

"You can do one gene and one mutation at a time,” he said. “Even with the CRISPR [gene editing] technology we have now, it still costs a huge amount of money and time and most labs cannot do it, so we wanted to develop something simple every lab can do.  No other approach is so efficient and fast right now.”

In addition to ramping up the velocity of studying gene mutations, the new approach may be able to reduce failures in the research process by giving researchers a more sensitive, targeted way to stimulate gene activity. 

"The problem in the cancer field is that they have many high-profile papers of clinical trials [that] all failed in some way," Zhu said. "We wondered why in these patients sometimes it doesn't work, that with the same drug some patients are getting better and some are getting worse. The reason is that you don't know which drugs are going to help with their particular mutation. So that would be true precision medicine: You have the same condition, the same syndrome, but a different mutation, so you have to use different drugs."

Zhu has already used the method to analyze approximately 50 mutation of the BRaf gene, which has been tied to tumor development and certain neurodevelopmental disorders.  He envisions that the technique will also help unlock the secrets of other diseases, such as Alzheimer’s, cystic fibrosis, and a variety of cancers – all of which are top priorities for precision medicine researchers.

As the marketplace for targeted therapies and associated precision medicine technologies approaches the $100 billion mark, techniques that can help cancer researchers accelerate the development of new treatments will continue to be in high demand. 

Drastically reducing the time from hypothesis to bedside will likely produce financial benefits for research labs as well as clinical benefits for patients.

“You'd need to spend 10 years to do what we are doing in three months, so it's an entirely different scale,” said Zhu. “Now, hopefully, we can do 40 or 100 of them simultaneously."


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