Gene therapy and genome editing for lipoprotein disorders

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Paper Summary

Conflicts of Interest

Identified Weaknesses

Rating Explanation

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Paper Summary

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Gene Editing Therapies Show Promise for Lowering Cholesterol, But More Research is Needed

This review discusses the potential of gene therapies, specifically gene addition and gene editing, for treating lipoprotein disorders like familial hypercholesterolemia and elevated lipoprotein(a). While promising results have been seen in preclinical and early clinical trials, highlighting the potential for durable treatments that could improve adherence, further research is needed to address challenges such as off-target effects, immune responses, and the high cost of these therapies.

Possible Conflicts of Interest

Several authors disclose financial ties to pharmaceutical companies involved in developing gene therapies for lipoprotein disorders. For a detailed list of disclosures, please refer to the 'Declarations' section of the paper.

Identified Weaknesses

Early stage of research

Many of the discussed therapies are in preclinical or early-stage clinical trials. Long-term efficacy and safety in humans are still unknown.

Limited long-term data

The durability of the gene editing effects and the long-term safety profiles need further investigation, particularly regarding potential off-target effects and immune responses.

Cost

Current gene therapies are extremely expensive, which could limit accessibility for many patients.

Delivery challenges

Efficient and targeted delivery of gene editing tools to the desired tissues remains a challenge, particularly beyond the liver.

Rating Explanation

This review provides a comprehensive overview of a promising field with real potential to improve treatment of lipoprotein disorders. However, the rating is capped at 4 due to the early stage of research for many of the discussed therapies and the need for more long-term safety and efficacy data in humans.

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