"Gene-Editing Treatment: A Promising Solution for Lowering High Cholesterol"

1 min read
Source: WIRED
"Gene-Editing Treatment: A Promising Solution for Lowering High Cholesterol"
Photo: WIRED
TL;DR Summary

Researchers have conducted a small-scale trial in which a single infusion of a gene-editing treatment successfully lowered cholesterol levels in patients with an inherited condition causing high LDL cholesterol. The treatment, which uses a newer form of Crispr called base editing, aims to permanently reduce cholesterol by inactivating a gene in the liver. Preliminary results showed that the highest dose of the treatment reduced LDL cholesterol by 55% in one patient, while the next-highest doses resulted in reductions of 39% and 48% in two other patients. The therapy could potentially provide a lasting alternative to long-term medication for hereditary high cholesterol, but safety concerns and potential off-target effects of gene editing remain important considerations.

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