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Local experts discuss latest advancement in gene therapy

DNA rendering

CRISPR is a tool that allows one to go into a person’s genome–which is the complete set of genes in a cell–and manipulate it to add, subtract or change a person’s genes.

“Say you have a disease mutation within your genome, you can change that disease mutation back to a non-disease state,” said Heidi Hehnly, an assistant professor of biology at Syracuse University. In her lab, she uses CRISPR to understand the molecular process in which cancerous cells, among others, divide.

The genome-editing tool, CRISPR, has been used in labs, like Hehnly’s, for years and has even recently been used to treat blood-based diseases like Sickle Cell Anemia. However, now, scientists have made their biggest advancement yet.

In June, data was reported in the New England Journal of Medicine that proved CRISPR was successfully used to treat Transthyretin Amyloidosis–a rare and fatal hereditary disorder that causes a buildup of misshapen proteins in the body which cause destruction of vital tissue.

“What makes this so different is that the CRISPR molecules were directly injected into the patient, they went to the place where they needed to go, made the genetic change and led to a safe and effective treatment for the disorder,” said Dave Dunn, an associate biology professor at SUNY Oswego.

He said this advancement in gene therapy is transformative for medicine.

“This is the dawning of a new age in gene therapy. And we're seeing CRISPR as the Great Leap Forward,” said Dunn.

Hehnly said that while she’s thoroughly impressed by the latest CRISPR advancement, she doesn’t see it being used in normal medical practices for a long time.

“It's going to be hard to really introduce it into medicine quickly because I think the ethics of CRISPR–it needs to be addressed and made sure that we're using this technology appropriately,” said Hehnly.

The common ethical concerns surrounding gene therapy include the fear of artificial selection or using CRISPR to delete undesirable traits from people’s genomes.

“Most new technologies have the capacity to do incredible good, but they also have the capacity to be used for ill,” said Dunn.

Regardless, both scientists are optimistic about how monumental this advancement will be for the way we can combat disease.

“I just think it's going to be revolutionary for medicine,” said Hehnly.