PROJECT SUMMARY/ABSTRACT
The development of new methods for prenatal genetic editing in humans has accelerated the translation of
technologies designed to disrupt disease mechanisms or remove disease-causing mutations entirely. These
technologies show promise for preemptively addressing previously intractable genetic conditions. However,
recent events have demonstrated considerable gaps in the governance structure surrounding human gene
editing and its translation to clinical practice. Since the discovery that a scientist in China had conducted human
experiments allegedly resulting in the birth of three genetically edited children, numerous calls have been made
for a novel governance structure for prenatal human gene editing, frequently framed around moratoria or other
forms of suppression, and several international bodies have convened. There has been strong pressure towards
a values-based governance approach that moves beyond traditional technocratic considerations of safety and
efficacy and takes into account collective normative deliberation about the ethics of prenatal intervention. This is
challenging in the United States context, which is among the few high-income countries without centralized
regulation of research on embryos and/or translational assisted reproductive technologies. As such, two
important stakeholder voices have been largely left out of conversations around the future of prenatal gene
editing in the US context: the patients and families that might be benefitted or harmed by the translation of
prenatal gene editing and the scientists and clinicians who would be on the front lines of clinical translation were
it to move forward. We propose to fill this gap by conducting empirical research with these key stakeholders that
assesses potential governance approaches internationally and explores how they may be implemented in an
United States context. The goal is to move past generalizations to explore the direct policy mechanisms that are
feasible while incorporating the values and priorities of end users.
This study consists of three aims. The first two, contemporaneous, aims will consist of qualitative research with
two sets of stakeholders: patients and families affected by genetic conditions potentially addressable through
prenatal gene editing and clinicians and scientists involved in relevant translational and clinical activities in this
space. In Aim 1, We will begin by conducting a review of the international science policy landscape to gather
policy mechanisms that have been proposed or implemented to manage emerging technologies in the
reproductive science, genetics, and regenerative medicine spaces. We will identify policies that fall into a
spectrum from permissive to restrictive. These policies will be shared with stakeholders during qualitative
interviews to assess their concordance with stakeholder values and priorities. We will recruit a diverse cohort of
clinicians and scientist leaders from 20 key professional societies and conduct qualitative interview to assess
underlying normative underpinnings of proposed policy approaches, professional feasibility of proposals, and
values and priorities around the potential translation of prenatal gene editing in humans. The results of this Aim
will feed directly into the deliberative democracy exercises in Aim 3. In Aim 2, we will partner with Genetic
Alliance, the largest patient advocacy organization for genetic conditions to recruit a panel of patient advisors
from communities affected by 9 genetic conditions from across a spectrum of penetrance and severity. Together
with the advisors we will connect with patient advocacy and support groups in these communities to recruit a
cohort of patients and family members. Patients will be asked to share their understanding of the potential of
gene editing, their aspirations or concerns about its clinical applications, their priorities and goals in research on
their condition, and their views on the proposed policy approaches. In Aim 3 we will convene representatives
from both groups, together with the research team and select policymakers to conduct two deliberative
democracy exercises in which all stakeholders can deliberate towards a consensus on the appropriate
mechanisms for optimal governance of prenatal gene editing technologies. The results of all three aims will be
disseminated in the scientific, lay, and policy literatures.