Latest News About Gene Knockout

Updated 2026-05-12 10:04

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How Human 'Gene Knockouts' Are Pointing the Way to New Medicines

Some people are naturally missing the working copy of a gene, the result of which can range from no effect at all to, occasionally, profound effects, such as very low cholesterol or congenital insensitivity to pain. Such significant, unusual medical characteristics are commonly referred to as “extreme phenotypes.” These extreme phenotypes and their underlying genetic causes represent, in a sense, experiments of nature that point the way toward a new understanding of disease, and potentially...

www.pfizer.com

Human Knockout Project

Naturally occurring human genetic variants provide “experiments of nature” that can directly inform on the function of human genes. In addition to providing novel insight into human biology, they can aid in the identification and validation of genes that would be powerful and well-tolerated targets for therapeutic inhibition in both rare and common diseases.

www.broadinstitute.org