By: Maggie Lynch

Engineers at Duke University have spent years trying to perfect their solution to gene editing and they believe they have finally done it.

CRISPR is a bacterial technology that scientists have adopted in recent years to conduct gene editing and modification. With this tool, they are able to delete and replace specific sections of a DNA strand. As it currently exists, CRISPR technology is mostly used on DNA modification of single celled organisms or early embryos of mice for testing.

However, the ultimate goal, for some scientists, is DNA modification in humans. Presently, some researchers are experimenting with stem cell modification to aid in the recovery of sick and healing patients.

The Problem

In order to ensure safety of gene editing, the technology itself had to be improved. Scientists around the globe have been working towards this goal for years, all failing to fully cleanse the technology of certain defects. Generally, the solutions thus far have all produced unwanted genetic edits in addition to their intended modifications and have been non-transferrable amongst the various CRISPR systems.

Enter Charles Gersbach and Dewran Kocak: professor and PhD student at Duke University’s Biomedical Engineering school. Together, the two seem to have found the solution to the CRISPR technology. Their goal was to develop a solution that would be usable across all CRISPR systems and that would not require the addition of any parts, but rather focus on the tools already in use.

“CRISPR is generally incredibly accurate, but there are examples that have shown off-target activity, so there’s been broad interest across the field in increasing specificity,”  

Charles Gersbach, the Rooney Family Associate Professor of Biomedical Engineering at Duke

The Solution

Their solution involves adding a short tail onto the end of the RNA sequence which will then fold back into itself because of the extra length, forming a lock in the shape of a hairpin. The lock can only be broken when the RNA pairs with the correct DNA sequence to properly modify the strand.

Thus far, Gersbach and Kocak have only tested their findings on cultured cells, but in the coming months they hope to begin trials using animals to yield more accurate results. This tactic, and the long-term goal of human DNA modification, brings up the issue of ethics in biomedical engineering.

“What’s common to all CRISPR systems is the guide RNA, and these short RNAs

are much easier to engineer.”

Dewran Kocak

A CRISPR Future

As in all fields of science, engineering has often crossed ethical boundaries in order to discover life-changing cures and methods. However, where do we draw the line with gene modification? This technology has the ability to progress into one that allows for “customizable babies”: the futuristic idea by which parents can select certain genes for their unborn children.

Currently, testing with human embryos is highly regulated worldwide but specific regulations differ between countries. While the whole idea of customizable babies is still very far off, creating any sort of technology that aids in the ability to genetically modify a human being is a major concern for some ethics committees. The CRISPR technology and its advancements will be of high importance to the science community in the coming years.