CRISPR gene editing offers a potential alternative

CRISPR gene editing offers a potential alternative.

A new study by Intellia Therapeutics aims to treat hereditary angioedema using CRISPR technology. Bradykinin is responsible for vascular permeability, where significant increases in bradykinin caused by stress, illness, or trauma leads to severe, painful, and life-threatening swelling. The molecular mechanisms that drive the angioedema seen in the limbs, abdomen, and throat of individuals affected by the disease are caused by mutations that produce a dysfunctional protein called C1-esterase inhibitor, which controls the levels of bradykinin in the blood.

Therapeutic strategies that treat hereditary angioedema work by blocking kallikrein, a protein that upregulates the production of bradykinin. Since hereditary angioedema is a lifelong chronic disease, people suffering from continual swelling will have to take kallikrein inhibitors for life.

CRISPR gene editing offers a potential alternative. The Intellia study enrolled 3 individuals, all received a low dose of the CRISPR lipid nanoparticle and all three saw a 65% decrease in kallikrein at 8 and 10 weeks. An additional 3 patients were administered a higher dose of the CRISPR lipid nanoparticle, and a 92% decrease in kallikrein was observed.

All patients prior to treatment were experiencing severe swelling 3-7 times a month. After CRISPR therapy, no swelling was experienced, and all patients were able to stop the drugs they were taking to prevent swelling. The ability to intravenously inject CRISPR technology instead of ex vivo gene editing allows for a broad range of therapeutic applications where cures for hereditary genetic diseases were once fairy tales you told your children at bedtime.

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Canan Schuman, PharmD/PhD
Author: Canan Schuman, PharmD/PhD

Canan Schumann is Chief Editor for Axxiem and for Axxiem's blog "BiotechOntheWeb". When not writing for Axxiem, Canan works as a Clinical Research Scientist II at the Research and Development Department at Molecular Testing Labs, developing endpoint assays for the detection of infectious disease and cancer. Canan currently resides in Portland, Oregon, where he received his Honors Bachelor of Science (HBS), Doctor of Pharmacy (PharmD.), and his Doctor of Philosophy (Ph.D.) in Biopharmaceutics at Oregon State University.