CRISPR-Based Gene Therapy Lowers Cholesterol

Experts have recently introduced a single-use CRISPR-based gene therapy to reduce levels of “bad” cholesterol in a small trial involving people with familial hypercholesterolemia (FH), a genetic condition that increases the risk of heart disease. This gene therapy, called VERVE-101, has shown promising results in reducing low-density lipoprotein (LDL) levels in the blood.

Potential Long Wait for Approval

While the new treatment shows potential benefits, it may take a long time before it is approved for use in patients.

The Role of Mutations and Gene PCSK9

FH is caused by mutations in a key gene, PCSK9, which affects the liver’s ability to remove LDL from the blood. This gene becomes overactive in people with FH, leading to a significant increase in cholesterol levels, requiring cholesterol-lowering medications to manage.

VERVE-101 and CRISPR Technology

The gene therapy uses a modified version of CRISPR technology called base editing, which can precisely modify DNA sequences, reducing the risk of unintended mutations.

Preliminary Results from Clinical Trial

Preliminary data from the ongoing clinical trial showed that patients who received different doses of VERVE-101 had varying responses. Some saw a decrease in LDL levels, while others did not respond to the treatment at all. However, two participants experienced severe heart problems during the trial, with one participant in the low-dose group passing away due to cardiac arrest.

Importance of Safety

Dr. Carol Watson, a cardiologist involved in the trial, emphasized the importance of safety in gene-editing studies, particularly as there are already safe and effective cholesterol-lowering strategies available.

Source: Live Science