Human Genome Therapy Jennifer Yang/Getty Images

In Defense of Germ Line Gene Therapy

Human gene therapy has been one of the most ambitious goals of biotechnology since the advent of molecular techniques for genetic modification in the 1970s; but it has also been highly controversial. With the technology now reaching a milestone, discussions about its applications have reached fever pitch.

Editors’ Note: August 4, 2017
Legitimate objections have been raised about the independence and integrity of the commentaries that Henry Miller has written for Project Syndicate and other outlets, in particular that Monsanto, rather than Miller, drafted some of them. Readers should be aware of this potential conflict of interest, which, had it been known at the time Miller’s commentaries were accepted, would have constituted grounds for rejecting them. 

STANFORD – Human gene therapy has been one of the most ambitious goals of biotechnology since the advent of molecular techniques for genetic modification in the 1970s. But it has also been highly controversial. With the technology now reaching a milestone, discussions about its applications have reached fever pitch.

Until now, only one approach – somatic cell human gene therapy – has been viable. SHGT alters a patient’s genes by editing existing genes or inserting new ones, in order to correct conditions present at birth or acquired later in life. Somatic cells are any in the body except eggs or sperm; thus, modifications made to them are not heritable. During the last quarter-century, SHGT has achieved several successes, including the correction of rare genetic abnormalities that cause recurring pancreatitis or blindness from degeneration of the retina.

But another approach – “germ line gene therapy” (GLGT), which, by modifying sperm, eggs, or embryos, creates a heritable change that affects future generations – is now also approaching practicability. Last May, Chinese researchers published the results of a partly successful proof-of-principle attempt to edit genes with a system called CRISPR-Cas9, using nonviable embryos that were going to be discarded in any case.

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