Editorial
Safeguarding the future of human gene editing
In his 1978 book The Beginning and the End, Isaac Asimov
wrote, The advance of genetic engineering makes
it quite conceivable that we will begin to design our
own evolutionary progress. Nearly 40 years ago, the
enormous prospect of human gene editing was but
a twinkle in Asimovs eye, but today, it has become
perhaps the most promising, and controversial, frontier
of modern medicine. And indeed, as predicted, one with
the potential to potently inuence human progress.
Because of the vast promise that gene editing holds
and in anticipation of the numerous ways in which that
promise and patients must be protected, the National
Academy of Sciences and the National Academy of
Medicine convened the Committee on Human Gene
Editing: Scientic, Medical, and Ethical Considerations.
The committee has spent the past 2 years examining
in-depth the clinical implications and social, ethical, and
legal ramications of human gene editing technologies.
Culminating in a report released on Feb 14, 2017, Human
Genome Editing: Science, Ethics, and Governance is a broad
framework by which developments in this rapidly
evolving and advancing eld can be assessed.
The capability to edit the genome by altering DNA
sequences has existed for some time. It has previously
been used in various capacities in non-human
organisms, such as enhancing crop seeds and laboratory
animal engineering, but the technology supporting it
continues to be improved and rened. Most recently,
CRISPR/Cas9 has garnered substantial attention for
being a cheaper, faster, and more precise gene editing
tool. CRISPR (clustered regularly interspaced short
palindromic repeats) are short RNA sequences used with
Cas9 (CRISPR-associated protein 9) or similar exogenous
nucleases, that when delivered to a cell, can cut the
genome to excise DNA sequences or introduce new ones.
This molecular scissor approach has introduced a
surge of research into new biomedical applications for
treatment and prevention of disease. But with such
developments, this wave of gene editing technology has
also ushered in a set of more clearly articulated ethical
questions. The report places considerable emphasis on
the important distinction of the class of target cells
somatic or germline. Most basic biomedical research
is done with somatic (non-reproductive) cells and, as
such, the risks and ethical concerns are conned only to
www.thelancet.com Vol 389 February 18, 2017
the individual. Because the CRISPR/Cas9 system shares
many features with other types of gene therapy, the
safety and regulatory infrastructure that is already in
place might provide a sucient foundation for rapidly
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moving the technology into clinical settings.
Alternatively, editing germline cells opens up an
entirely dierent set of unknowns and ethical quandaries.
Germline editing has great appeal in that it would
seemingly harness the ability to correct genetic
mutations present in an individuals genes before
those genes are passed to ospring, with the promise
of eradicating heritable conditions or diseases. But that
promise is imperilled with daunting ethical issues. Not
only is it impossible to know some of the ways in which
viable ospring might be aected, but the limitations
of current technologies would necessitate discarding
embryos or selective abortion when the editing has not
been successful. A second editing quagmire, and one
that could also apply to somatic cells, revolves around
the notion of enhancement as opposed to correcting a
detrimental mutation. Although somatic enhancement
might have therapeutic value for an individual,
enhancement of traits or capacities through germline
editing raises questions about eugenics and makes salient
a host of undesirable repercussions, such as the societal
treatment of individuals with disabilities to unfair access
to such technology on the basis of socioeconomic status.
The committee makes recommendations for moving
ahead with human genome editing by promoting
wellbeing, respect, and fairness, in addition to adhering to
the principles of transparency, due care, and responsible
science to preserve ethically responsible research and
clinical use. Importantly, though the recommendations
reference US regulations, transnational cooperation will
require open dialogue between countries, regulatory
agencies, and scientic communities, a reality that may
be easier to conceive than to achieve. Still, the report
comes at a crucial time. With the rst clinical trial using
CRISPR targeting cancer cells approved in the USA in
June, 2016, and the rst CRISPR-edited genes injected
into a patient in China in November, 2016, the race for For the report Human
Genome Editing: Science,
results is under way. Although, for now, the report signals Ethics, and Governance
both the beginning of a new phase of productivity and see https://www.nap.edu/
catalog/24623/human-
an end of sorts to the debate around the way forward for genome-editing-science-
human gene editing. The Lancet ethics-and-governance
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