Is Medicine Ready for Clinical CRISPR?

Using precision genome editing to treat or prevent human disease may require several leaps of faith.

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OXFORD UNIVERSITY PRESS, AUGUST 2016CRISPR-Cas9 genome editing has the potential to transform medicine in several important ways. First, the technique makes it possible to manipulate genes in a variety of mammals to create models of human health and disease. Previously, only mice could be engineered in this way, but genome editing has made it possible to precisely modify the genomes of almost any mammal.

Because pig hearts or monkey brains are far more similar to their respective human organs than those of mice, this should have a major impact on our ability to understand the genetic basis of heart disease and various mental disorders. But such developments are likely to be controversial because of opposition by some people to experimentation on primates.

Another way that genome editing affects medicine is by facilitating the study of physiological or pathological processes in human cells in culture. Using genome editing to precisely manipulate the genomes of human cells in vitro is making it possible to identify the genes involved in normal human physiology and in various types of human disease. I discuss these and other transformative applications of CRISPR-Cas9 genome editing in ...

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