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Heavy lifting ahead if the U.S. is to do heritable genome editing - STAT C 1 The U.S. must do some heavy lifting to prepare for heritable genome editing By Josephine Johnston September 4, 2020...

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Heavy lifting ahead if the U.S. is to do heritable genome editing - STAT
The U.S. must do some heavy lifting to prepare for heritable
genome editing
By Josephine Johnston
September 4, 2020
ountries contemplating giving the green light to heritable genome editing
eceived specific guidance3 from an international commission this week on
how to prepare for a future in which the technology is safe and effective
enough to use in human reproduction.
The commission was created in response to the news4 almost two years ago
that a scientist in China had edited the genomes of two babies5 when they were
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single-cell em
yos. (It subsequently emerged that a third baby with an edited
genome had been born.)
While the scientist involved, He Jiankui, was widely condemned fo
conducting a premature and unethical experiment, and later fined and
sentenced to jail6, his first-in-humans experiment pushed the international
community beyond a “germline editing — good or bad?” discussion to actively
considering what it would take to justify its use.
Germline gene editing raises particular ethical and safety issues. Gene editing
in a young child or adult creates changes that may affect the individual, but are
not passed on to his or her children. Germline editing, also known as heritable
genome editing, creates changes that are inherited by subsequent generations.
Related: 7
Expert panel lays out guidelines for germline editing, while
warning against pursuit of ‘CRISPR babies’ 7
The International Commission on the Clinical Use of Human Germline
Genome Editing, an expert committee co-sponsored by the U.S. National
Academy of Medicine and National Academy of Sciences and the United
Kingdom’s Royal Society, was specifically charged with defining a pathway to
using CRISPR or other gene editing tools to modify germline cells (sperm,
eggs, and their precursors) or very early-stage em
The committee begins its report by stating that the technology is not yet
sufficiently safe to use in humans and that, even if it were, the decision whethe
to permit it “must ultimately rest with individual countries.” Yet the very
existence of the committee, and the nature of its recommendations, recognize
the promise of this technology and that a strong case can be made for its use by
prospective parents “with a known risk of transmitting a genetic disease” o
“with reduced fertility.”
In the committee’s 11 thoroughly reasonable recommendations, it proposes an
incremental path forward, beginning with the use of genome-editing
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technologies to prevent serious diseases caused by mutations in a single gene
(monogenic diseases) such as Huntington’s disease, cystic fi
osis, and sickle
cell disease, that cause severe mo
idity or premature death.
As reasonable as the recommendations are, they will require work by interested
nations to meet the commission’s proposed preconditions for using heritable
human genome editing. The challenges for U.S. health and technology policy
are especially significant. Here are three that will be heavy lifts for the U.S.:
1. The report recommended that countries undertake “extensive societal
dialogue” before making any decision about whether to permit heritable
editing. While some countries have a history of extensive public engagement
around controversial new technologies like genetically modified foods8, no
such dialogue has been attempted in the U.S. regarding genome editing, or any
other emerging technology for that matter. It is hard to imagine cost-effectively
engaging large swaths of the American public on this issue or why doing so
could be considered a higher priority than engaging them on other more
pressing issues, such as pandemic recovery, racial justice, or political
A more modest proposal might suffice, such as targeted engagement with
specific stakeholders and deliberation with a small but representative sample9
of the public. But even that would be departure from America’s existing
approach to policy in this area, which thus far amounts to a de facto ban on
heritable genome editing enacted via a budget rider10 first passed in 2015
whose merits were not initially publicly discussed and which has, since then,
een annually re-upped with little to no debate11.
Related: 5
The CRISPR shocker: How genome-editing scientist He Jiankui
ose from obscurity to stun the world 5
2. An early emphasis in the report is that heritable editing, like othe
genetic technologies, should be deployed in ways that avoid bias and
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discrimination and “ensure equitable access.” An immediate question is
how the U.S. can satisfy this precondition when it is almost entirely alone
among developed nations12 in not providing universal access to health care?
In its recommendations about how countries should decide who merits access
to heritable editing, the commission requires that prospective parents first try in
vitro fertilization with preimplantation genetic diagnosis13, reproductive
technologies that many American health plans do not cover. If made available
today, the new genetic technology the report focuses on would, like so many
others, be available only to those Americans with exceptional insurance
coverage or the ability to pay out of pocket. Significant changes to U.S. health
policy would be needed to meet the “equitable access” recommendation fo
heritable genome editing.
3. The commission calls for countries to have mechanisms and regulatory
odies in place that allow for prospective and ongoing use-by-use
assessment of the safety and ethics of all applications of heritable editing.
The U.S. Food and Drug Administration could assess the safety and
effectiveness of this technology if it was authorized to do so, and institutional
eview boards could protect human research subjects.
But the U.S. does not have in place a regulatory structure for governing
assisted reproductive technologies like in vitro fertilization, which in countries
like the United Kingdom and Australia can be adapted to accommodate
heritable editing.
The American Society for Reproductive Medicine and the Society for Assisted
Reproductive Technologies, professional organizations that represent fertility
clinics and providers, issue guidance addressing clinical and ethical issues in
fertility medicine, such as egg freezing and sex selection, and could address the
use of heritable genome editing. But their guidance is voluntary and
notoriously permissive14, allowing procedures that other countries ban, and it
is unlikely to satisfy the commission’s requirement for a national oversight
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The report acknowledges that the world’s attention is appropriately focused
today on the “twin upheavals” of the devastating Covid-19 pandemic and this
summer’s urgent calls to address racial injustice and inequities. While the
prospect of heritable genome editing is very different than those two pressing
issues, the committee points out that it also transcends individual countries,
deserves wide-ranging discussion, and raises important equity implications.
That is exactly right. And it is especially striking at this moment in U.S. history
that in order to prepare for heritable editing — at least according to the
ecommendations of this commission — the U.S. would need to make health
and technology policy changes that would democratize science policy, secure
universal access to health care including fertility treatment, and create a
easonable regulatory framework for reproductive medicine. A relatively small
number of Americans families with serious heritable genetic diseases are likely
to seek out germline editing. Yet making the changes to U.S. policy to give
them the opportunity to do that will benefit everyone.
Josephine Johnston is director of research and research scholar at the
Hastings Center and co-editor of the book “Human Flourishing in an Age of
Gene Editing”15 (Oxford University Press, 2019).
About the Author Reprints2
Josephine Johnston
@bioethicsjosie 17
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After the Storm — A Responsible Path for Genome Editing

n engl j med�� 1
The recent announcement of the birth of twins whose genomes were edited during in vitro fertilization (IVF) has engendered
oad con-
demnation for the premature clinical deployment of
a fledgling but powerful biotech-
nology. The Chinese scientist He
Jiankui may claim priority for the
first use of CRISPR-Cas9 genome
editing in human em
yos, but he
will forever be remembered for his
eckless flouting of widely artic-
ulated scientific, clinical, and eth-
ical standards.
CRISPR-Cas9, which first ap-
peared in the scientific literature
only 7 years ago,1 is a programma-
le, sequence-directed, RNA-guid-
ed nuclease whose rapid assimi-
lation has transformed biomedical
esearch and promises clinical ap-
plications in gene-repair strategies
to treat disorders such as sickle
cell anemia, congenital forms of
lindness, and muscular dystrophy.
The existing regulatory framework
for somatic gene therapy using vi-
al vectors has been honed through
decades of trials and should be ef-
fective at balancing scientific rigor,
patient safety, and innovation for
trials of somatic genome editing.
Treating disease by genome edit-
ing of somatic tissues raises few
ethical concerns, as long as patient
welfare is prioritized. The promise
far outweighs the medical risk.
Genome editing of early human
yos raises far greater con-
cern because it can lead to herit-
able changes in the germline. But
though the world’s first applica-
tion of em
yo editing was deeply
flawed, not least because it didn’t
address an unmet clinical need,
and has prompted calls for a mor-
atorium or an outright ban, we
elieve that halting research and
deliberation on more responsible
ways to maximize human benefit
would be unwise.
yo editing poses scientific
and ethical hurdles. In 2015, after
the first report that genome
Answered 1 days AfterMar 17, 2022


P answered on Mar 19 2022
68 Votes
Solution 1:
HRPP is a human research protection program with the aim in protecting the humans by better understanding and implementation of various treatments and programs which are involved in improving the health of the individuals besides minimizing the risk of the individuals and the subject’s equity regulations [1]. The advances in the technology has created a new approach in the biotechnology for the effective cure of genetic disorders and is being studied for the diagnosis of the rare and uncommon diseases by which the life span of the individual will increase. Gene therapy and cell therapy are the most advanced studies which were being studied by the researchers for a better healthy society [2]. Most of the researchers are using these gene editing techniques to cure the serious birth defects of the individuals [3]. There are some researchers who are supporting the moral necessity of these gene editing techniques and some argue that it is no necessary of gene editing techniques instead these can be overcome by the alternate technologies like in vitro fertilization (IVF), pre-implantation genetic diagnosis (PGD) and termination of pregnancy (TOP) for improving the health of the individual. However, there are limitations as well as the advantages for the different technologies which are cu
ent in use as well as for the technologies like the gene editing which are under trials [4].
The advantages of the gene editing techniques are:
1. Can be potential theurapeutical tool for diagnosing genetic disorders and chronic diseases like cancer and diabetes [4].
2. Polygenetic disorders which can’t be cured by PGD can be diagnosed by Gene editing techniques [4].
3. Disease ca
iers can be eliminated by Gene editing techniques which protects the future generations [4].
1. Sometimes unpredictable effects can be seen by the advances in the technologies. Ex: Cyberte
orism [4].
2. May lead to off-target mutations [4].
However, there is a need to control the technologies by the law which can minimize the future catastrophic events as well the off-target mutation leading to the disabilities in the individuals [5]. It was understood form the literature that there is a global agreement where the individuals should be minimally harmed i
espective of the less chances of the benefits [4].
One of the most success research of the genetic engineering study on increasing the lifespan of the Methuselah mice was reported by Bartke hence, the same study can be implied to the humans for increasing the lifespan [6]. All these studies should be conducting by following the ethical procedures i
espective of law and the science to protect form the misuse of this technology. Besides the limitations, it is very moral for the application of the gene editing techniques if it is used in a proper way under the guidance of the regulatory authorities to minimize the burden of the genetic disorders [4].
Yes, gene editing technology is a novel strategy in comparison to the other clinical research...

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