2019-11-04_Time

80 Time November 4, 2019

HEALTH CARE • VIEWPOINTS

ever since The discovery of The sTrucTure of dnA in
the 1950s, scientists have been dreaming about rewriting the
code of life. What if we could correct genetic mutations that
cause disease in order to radically improve human health?
Harnessed from the naturally occurring immune system that
bacteria use to defend themselves against viruses, CRISPR-
Cas9 is a revolutionary, once-in-a-
generation tool that offers the real
potential to quickly and efficiently
achieve what was once thought
impossible.
Since 2012, the technology has
been adopted rapidly, transform-
ing basic research, drug develop-
ment, diagnostics and agriculture.
In the past seven years, over 15,000
papers containing the term CRISPR
have been published, and hundreds
of different organisms have been
edited. CRISPR has become a main-
stream topic of conversation, fod-
der for Hollywood scriptwriters and
the standard genome- editing tool used globally. As we move
into a new decade, it is clear that CRISPR- based applications
will help us tackle societal challenges including disease, food
production and environmental sustainability.
I receive daily emails from people suffering from debilitat-
ing genetic diseases asking how and, especially, when CRISPR
can fix what is hardwired in their DNA and often runs in their
family. For many diseases, like Huntington’s and Tay-Sachs,
we know the gene that causes the disease but have so far been
power less to change it. But now, thanks to the CRISPR revo-
lution, we can shift the paradigm entirely. Sickle-cell research
at the Innovative Genomics Institute, where I am executive

The gene-editing

revolution is

already here

By Jennifer Doudna

Ensuring

responsible

use of

CRISPR is

an ongoing

challenge

director, and elsewhere shows we can pro-

actively mitigate or correct the mutation that

causes the illness. Gene- editing treatments for

this and other diseases are beginning or will

soon enter clinical testing. The gene- editing

revolution has led to a rapidly growing CRISPR

economy, and over the next decade, the tech-

nology will likely produce tangible and poten-

tially wide- ranging treatments and even cures

for genetic diseases.

For all the promise of CRISPR, ensuring

responsible use is an ongoing challenge. Al-

most a year ago, scientist He Jiankui shocked

the world by revealing that he had edited the

embryos of twin girls. It was a medically un-

necessary experiment that radically broke

the global consensus that CRISPR should not

currently be used in clinical human- germline

editing— that is, making genetic changes that

can be passed down to future generations. The

scientific community responded by redoubling

efforts to establish stronger safeguards, encour-

age a more deliberate approach and deepen

public conversation about responsible use.

The World Health Organization is now push-

ing government regulators to act. We need com-

pliance, not a moratorium, as the former invites

conversation, which is critical since interest in

editing the human germline is not going away.

During CRISPR’s teenage years, we will look

to expand the types of edits we can make, focus

on advancing the safe and effective delivery

of CRISPR genome- engineering tools, work

through the first wave of Food and Drug Ad-

ministration approvals and increase our explo-

ration of a naturally occurring way to fine-tune

CRISPR-based editing to improve accuracy.

There’s a possible future where genetic dis-

ease is a thing of the past, where we routinely

sequence DNA and treat harmful mutations as

an outpatient procedure. But we must ensure

that in this future, everyone will have access to

these new technologies and there’s a consen-

sus on rules to regulate whether and how this

technology is applied to the human germline.

This must come from a collaborative effort that

includes increased private and public invest-

ment, more commercial partnerships to reduce

financial risk and scale the technology, and the

political and regulatory nuance to allow wide-

spread affordable access to safe, effective cures

without stifling a technology that will under-

pin the health of future generations.

Doudna, a professor at the University of

California, Berkeley, co- discovered CRISPR-

Cas9 gene-editing technology

ILLUSTRATIONS BY JON STICH FOR TIME