Yhn HealthMental Health first seen 4 d ago, last 1 h ago, peak #1

One-Shot CRISPR Therapies Targeting PCSK9 Cholesterol Gene Advance

Original: In-Vivo CRISPR/Base-Editing "One-Shot" Therapies Targeting PCSK9

A new review in Current Atherosclerosis Reports examines in-vivo CRISPR and base-editing therapies that target PCSK9, a gene that regulates LDL cholesterol. The approach would require only a single infusion rather than lifelong daily medication, potentially offering a lasting reduction in heart attack and stroke risk. Early clinical programs such as VERVE-101 and similar candidates have drawn attention for permanent gene silencing, with debate over safety, off-target edits and who should receive irreversible treatment.

Why now: Interest in permanent, one-time gene-editing alternatives to statins as trial results for PCSK9-editing therapies emerge

PCSK9CRISPRVERVE TherapeuticsCurrent Atherosclerosis Reports

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Evidence

• In-Vivo CRISPR/Base-Editing "One-Shot" Therapies Targeting PCSK9 · brandonb · 9

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