Health
Gene-editing trial shows yearlong cholesterol reduction in small study
A updated pilot study in The New England Journal of Medicine shows a single CRISPR infusion cut cholesterol levels by roughly 50% over 12 months in four high-dose patients.
The short version
- An updated pilot study showed that a one-time gene-editing treatment sustained a roughly 50% drop in LDL cholesterol and triglycerides over a full year in four patients on the highest tested dose.
- The early-stage trial evaluated 15 individuals with severe high cholesterol that had not responded to traditional medications.
- Researchers reported no adverse effects in the four high-dose participants, suggesting the single-dose CRISPR-Cas9 infusion is safe and durable in early testing.
- Larger clinical trials are required to confirm the therapy's safety, efficacy, and long-term viability before it can become widely available.
Key facts
- A pilot study involving 15 patients evaluated five different doses of an experimental CRISPR-Cas9 gene-editing treatment for severe high cholesterol.[Slashdot]
- Four patients who received the highest dose experienced an approximately 50% reduction in LDL cholesterol and triglycerides that persisted for a year.[Slashdot]
- Researchers reported no adverse effects among the four participants in the highest dose group.[Slashdot]
- The updated trial findings were published in The New England Journal of Medicine by researchers affiliated with the Cleveland Clinic.[Slashdot]