CRISPR gene editing is in the news a lot these days as researchers use it to try to change the course of critical inherited diseases such as sickle cell or hemophilia. Recently, a biotech firm took CRISPR to the next level, creating a cousin to this innovative technology known as base editing.
What Is CRISPR Gene Editing?
CRISPR gene editing is a technology that can identify genome patterns in DNA and change them. This science is based in nature.
Bacteria infected with a virus can take small pieces of the invader’s DNA and insert them into their own. They use this to identify the virus should it invade in the future. The bacteria use RNA segments to recognize and bind to the virus if it returns. They then use an enzyme to attack the invader’s DNA and destroy it.
Scientists have taken this natural process and created technology to mimic it in humans. This allows them to identify strands of DNA patterns and make changes. The goal is to correct mutations that lead to diseases such as sickle cell and hemophilia. The current technology used in CRISPR Cas9 genome editing breaks both strands of the DNA’s double helix structure and stitches them back together. CRISPR gene editing is evolving into a less destructive process known as base editing.
What is Base Editing?
Base editing is similar to the current CRISPR gene editing, but it does not break both strands of the DNA. This process involves coupling a Cas9 protein to a second enzyme. The Cas9 protein helps direct the base-editing enzyme to the right place in the DNA. The base editing enzyme then edits a single base. This new process is being put to the test in New Zealand for a volunteer with heart disease.
Using Base Editing to Fight High Cholesterol
U.S. biotech firm Verve Therapeutics out of Cambridge, Massachusetts, is looking to use base editing to help correct a gene mutation that can lead to familial hypercholesterolemia, which is high cholesterol that runs in families. People with this condition are at risk for early-onset heart disease.
This study aims to change one adenine base to guanine in the DNA responsible for encoding the protein PCSK9. This protein plays a significant role in regulating blood cholesterol levels. Using base editing, they want to reduce the amount of PCSK9 produced and lower the levels of bad cholesterol that can clog arteries.
Disabling the PCSK9 protein is already done with medication, but that provides a temporary fix. With this new gene editing process, Verve Technology is aiming for a permanent fix for familial hypercholesterolemia. Base editing takes CRISPR gene editing to the next level to further target the technology.
An expert in the field at Amsterdam University Medical Centers calls the study very promising. Preclinical data already shows the treatment can reduce levels of PCSK9 by 81%, lowering blood cholesterol with no side effects. That is good news for those facing early heart disease due to this mutation in their DNA.
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