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This gene editing breakthrough could deliver hyper-specific cancer diagnoses



Nicholas Petrelli, director of the Gene Editing Institute and study author.
Photo: The Gene Editing Institute

Since researchers first discovered that bacterial immune systems can be manipulated to selectively alter the DNA of living things, CRISPR gene editing technology has been pushed through the boundaries of the cell wall limited. With CRISPR, scientists can excise and insert small pieces of DNA and even swap individual letters of the DNA to correct disease-causing genetic mutations. But – at least until now – all this cutting and pasting has occurred in cells.

A study published Thursday in CRISPR Journal shows how scientists at the Gene Editing Institute of Christiana Care Health System are published in Delaware CRISPR behind the barricade of the cell wall. They were able to rapidly make several changes to the genetic code by extracting DNA from human cells and placing it in a test tube, where a protein called Cpf1 penetrated the DNA and paved the way for CRISPR to process.

But why? do you want to do something like that? Previous CRISPR tools limit gene editing to short DNA snippets within a single gene. Extracting the DNA from the cell could allow multiple manipulations simultaneously.

In short, according to the authors of the study, this could make a valuable contribution to cancer diagnosis, to the replication of exact genetic mutations in the tumors of individual cancer patients, and to the precise identification of the type of cancer that a patient needs for targeted development. Researchers are already working on commercializing such a diagnostic tool. More importantly, however, the new technology could also pave the way for new gene-editing technologies that allow the removal and replacement of entire defective genes, not just small DNA snippets.

That could greatly enhance the usefulness of CRISPR. While the technology is promising to cure diseases such as sickle cell anemia, which is caused by a one-letter mutation, more complex diseases seem to be out of reach. This new technology could eventually change that.

But the breakthrough also addresses the breadth of CRISPR technologies that arrived in laboratories last year. The new tool relies on an enzyme known as Cpf1 instead of Cas9, the enzyme that is typically paired with the CRISPR system to cut DNA. The discovery of new CRISPR enzymes has helped to create a wealth of new uses for the technology. For example, while Cas9 results in blunt ends as it cuts through DNA, Cpf1 produces sticky ends that make it more suitable to remove larger chunks of the genetic code.

The ability to cut outside the cell, researchers said also reveal more about the secret of how CRISPR actually works to modify the genome.

Recently, work with various enzymes has led to breakthroughs, including editing of the epigenome instead of changes to the DNA itself and the use of CRISPR to diagnose disease.

Such studies point to the power and potential of CRISPR – not just as a tool to change the genome, but as a multifunctional powerhouse with applications we have not even imagined.


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