The Study
Patient Supported Approaches to Gene Editing (PASAGE) Study
Scientists are working on gene editing technologies to address a number of genetic conditions, ranging from severe conditions that are fatal in early childhood (e.g., some kinds of Spinal Muscular Atrophy, Tay-Sachs) to conditions that emerge in adulthood or increase risk of certain diseases (e.g., Breast Cancer, Alzheimer’s disease). There are also many conditions that impact each person and their life differently (e.g., Autism) and generate a range of views on gene editing for these conditions.
Currently, most gene editing-based therapies happen on clinical trials in adults, but it is expected that some will become available as treatments before birth. (This is different than editing embryos, which is not included in our project) There are many important questions about prenatal gene editing therapies, including what conditions this technology should be used for and who should be responsible for managing approval and access to gene editing therapies.
Because of this, it is important that we understand the views of people who might be impacted by these technologies.
We want to understand the views, ethical concerns, clinical aspirations, and policy needs from patients and families, clinicians, scientists, and policy makers so that we can develop a holistic understanding of their priorities and values.
Our study will talk to two groups of people to ensure that their voices are included in the development of prenatal gene editing:
Patients and families of people with the following genetic conditions:
Tay-Sachs
Spinal Muscular Atrophy Type 0-2
Sickle Cell Disease
PKU
Cystic Fibrosis
Huntington’s Disease
Hereditary Breast/Ovarian Cancer
Family History of Alzheimer’s
Hereditary Hearing Loss
Autism Spectrum Disorder
Physicians and scientists who work in:
Gene editing/therapy
Reproductive medicine
Genetics
Specialized Care for Individuals with Genetics Conditions
Members of professional scientific organizations with experience in policy making
Some questions we’ll ask:
Should prenatal genetic therapies be developed? For which conditions?
Who should decide which therapies are pursued? What does that process look like?
What is the risk/benefit conversation for prenatal gene editing? Who decided what is ‘too risky’?
How do we make sure everyone has equal access to potential therapies?
Our end-goal will be to bring together the values of all stakeholders (patients and families, physicians, scientists) to guide the medical and scientific communities if prenatal gene editing therapies are developed and become available.
