Project 3: Gene Editing for Visual Impairment

The NIH Common Fund has currently funded somatic cell gene editing projects through the external page Somatic Cell Gene Editing (SCGE) consortium, with the goal of translating gene editing into the clinic. At the December 2023 consortium kick-off, there were significant discussions about how the basic science grants could work towards a health equity focus so that as projects reach the clinical trials phase, they are well positioned to ensure that the ultimate translation of gene editing happens in a more equitable manner. The current project is a 1-year administrative bioethics supplement to a U19 grant “external page The CRISPR Vision Program: Nonviral Genome Editing Platforms to Treat Inherited Retinal Channelopathies” (external page PI K. Saha, University of Wisconsin-Madison) and will be conducted jointly between investigators at ETH-Zurich and the Univ. of Wisconsin-Madison. The proposal will both address bioethics capacity within the SCGE consortium and provide empiric data to address the specific ethical issues of health equity and justice in somatic cell gene editing for inherited retinal disorders (IRDs).  

Three aims

In Aim 1, we will perform a normative review of the health equity and justice issues for somatic gene editing therapies. This aim will increase bioethics capacity in the SCGE by providing a framework for future work within the consortium, and will also serve as a foundation for the empirical work in Aim 2.

In Aim 2, we will identify the ethical attitudes of patients/family members who have lived experience of retinal disease towards somatic gene editing, with a particular eye towards health equity and justice issues. This empiric bioethics project will include up to 30 interviews conducted in the United States (in English or Spanish) with persons who have a lived experience of IRDs. Topics covered in these interviews will include participant views towards (1) SCGE for IRDs and more broadly, (2) personal values, (3) individual and societal impacts of SCGE for visual impairments, (4) clinical endpoints, (5) consideration of potential risk and benefit across the translational pathway (clinical trials, early clinical availability), (6) information that they feel would be critical in order to provide informed consent for a future clinical trial or clinical administration of genome editing for IRD.

Finally, in Aim 3, we will integrate the experiences of Aim 1 and 2 to construct guidance for the other SCGE grantees that will help them consider the potential approaches toward community engagement and health equity in their future work.