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CRISPR for What Ails You

CRISPR-based therapies could someday be used to treat or even prevent common ailments, the Wall Street Journal writes.

Vertex Pharmaceuticals and CRISPR Therapeutics have been testing gene-editing approaches to treat conditions like sickle-cell disease and beta-thalassemia, while Verve Therapeutics has been exploring their use to modify high cholesterol and triglyceride levels in animal models. The Journal adds that CRISPR Therapeutics has further been developing an implant to provide edited cells that produce insulin to treat diabetes.

It adds that with tweaks to CRISPR techniques, such treatments could be used more widely. For instance, the Journal notes that a current effort to edit the PCSK9 gene in people with familial hypercholesterolemia could eventually have a broader application to people who have had heart attack or even as a tool to prevent heart attacks.

However, the Journal adds that there are not only financial and logistical issues to overcome, but also scientific challenges that need to first be addressed, including how to best deliver CRISPR machinery and determining how long such edits may last.