Dive into the microscopic world within us, where groundbreaking gene editing is poised to revolutionize heart health! In a groundbreaking clinical trial by Verve Therapeutics in New Zealand, a volunteer has become the first person to undergo DNA editing aimed at reducing blood cholesterol levels, a key factor in heart disease. This innovative approach uses a version of the CRISPR gene-editing tool to alter a specific part of the DNA within the patient’s liver cells. The goal of this precise genetic tweak is to permanently lower the levels of “bad” LDL cholesterol, which is responsible for the buildup of plaque in arteries, leading to heart disease and potentially heart attacks. In our AP Biology class, we learned that cholesterol is a type of lipid, or fat,  found in the cells of all animals. It’s essential for creating cell membranes, making hormones like estrogen and testosterone, and helping your body produce vitamin D and bile acids that digest fat. While cholesterol is crucial for these biological functions, too much of it, especially in the form of LDL (“bad” cholesterol”), can lead to health problems like heart disease. Cholesterol: friend, foe, or just misunderstood? Let us know down below!

The patient selected for this trial had a genetic predisposition to high cholesterol levels and was already experiencing heart disease. Verve Therapeutics believes that their gene-editing technique could be applied to a broader population to prevent cardiovascular diseases, the leading cause of death globally. The use of CRISPR technology for common conditions like high cholesterol represents a significant shift from its previous applications, which were mostly limited to rare genetic disorders. This approach could benefit millions who struggle to manage their cholesterol levels through conventional methods.

The treatment targets a gene called PCSK9, known to play a crucial role in regulating LDL cholesterol levels. By introducing a minor error in this gene through base editing, a more precise version of CRISPR that doesn’t cut the DNA but instead changes one DNA base into another, Verve aims to switch off PCSK9’s function. This interruption is expected to result in a significant and lasting reduction in LDL cholesterol, potentially preventing the development of heart disease in individuals with familial hypercholesterolemia (FH), a condition causing abnormally high cholesterol from a young age.

Protein PCSK9 PDB 2p4e

The technology behind Verve’s treatment is akin to the mRNA COVID-19 vaccines, utilizing nanoparticles to deliver genetic instructions to cells. This method directs liver cells to produce a base-editing protein that alters the PCSK9 gene, reducing LDL cholesterol levels. Early trials in monkeys have shown promising results, with a 60% reduction in bad cholesterol that has remained effective for over a year, indicating the potential for a permanent solution.

Cholesterol with numbering

However, the application of gene editing for cholesterol management is not without risks. Concerns include the toxicity of nanoparticles and potential side effects similar to those observed in other PCSK9-lowering drugs, such as muscle pain. Unlike traditional medications that can be stopped if adverse effects occur, gene editing is irreversible, presenting a challenge in managing unexpected outcomes.

Despite these challenges, the prospect of a one-time treatment for high cholesterol offers a revolutionary approach to combating heart disease. Verve’s gene therapy is anticipated to be more affordable than current gene therapies, thanks to the scalable manufacturing process similar to that used for COVID-19 vaccines. This advancement could make gene editing a viable and widespread treatment option, not only reducing the global burden of cardiovascular disease but also extending life expectancy by preventing heart attacks, the leading cause of death worldwide. Do you think that this techonolgy will be as promosing as it looks? Let us know down below!

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