New Gene-Editing Tool Greenlit for Cancer Trial in Humans

CRISPR (pronounced “Crisper”) is more than just a better way to prepare your bacon. In the genetics world, CRISPR stands for Clustered regularly interspaced short palindromic repeats. CRISPRs are found in prokaryotes as part of their immune system. They function by cutting exogenous (foreign) strands of DNA. But what we really care about here, is their function in gene editing in humans.

For all you Biochem geeks ou

For all you Biochem geeks out there, here’s the pathway

This process has become a hot topic in this field currently. By hijacking this system, we are able to incorporate genes into the genome of eukaryotes by inserting them through one of the many gene repair system that our cells have.

But what does all this mean?

Essentially, the majority of research in this field is being done in human cancers. In cancer, many genes are modified to be perpetually on or off. These are considered damaged and the hope is that with CRISPR, we will be able to change these genes and return them to normal functioning.

The current trial that has been approved is based of work done in UPenn which will use CRISPR to “teach” T-cells (the immune system’s attack dogs) to recognize cancer cells and leave healthy tissue alone. The hope is that it will allow our own immune system to properly recognize cancer and eliminate it. This work is focusing primarily on multiple myeloma but the hope is that it will be expanded to encompass a wide range of cancers.  In addition, if successful, there is potential for its use in HIV treatments as well.

Before we get too excited, the approval from this project came from the Recombinant DNA Advisory Committee (RAC). It still has to go through local Ethics Review Boards and the FDA.


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