Permanent cure? CRISPR combined with deactivated virus could solve ‘short-lived fix’ problem

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Gene therapy has shown promise in treating inherited genetic diseases, but a major issue that has frustrated scientists remains: Replacing a “bad” gene with a healthy one often is a short-lived fix. Typically, the healthy replacement gene works for just a few weeks. Now, scientists at Washington University School of Medicine in St. Louis have combined the gene-editing tool CRISPR with a deactivated virus.

The researchers ended the experiment at the six-month mark to study the mice, but said the length of sustained gene expression was equivalent to a fix that would last the lifespan of the mice.

[H]emophilia patients lack a gene that orchestrates blood clotting, leaving them at high risk of dangerous bleeding. A recent study of 10 patients showed that a virus (without CRISPR) was successful in delivering the missing gene and helping mitigate the patients’ symptoms.

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Scientists have no control over where the virus inserts the gene into the cell’s DNA code, raising the risk of undesirable mutations. But the advent of CRISPR gene-editing technology has changed that. While not perfect, CRISPR is a precise method to edit the genome and is user-friendly compared with other precision gene-editing techniques.

[Researcher David] Curiel and his team are further investigating the safety and efficacy of the combined adenovirus and CRISPR techniques, first in improving gene therapy for hemophilia.

Editor’s note: Read the full study

Read full, original post: CRISPR enhances gene therapy to fight inherited diseases

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