The Meaning of Informed Consent: Genome Editing Clinical Trials for Sickle Cell Disease.

ABSTRACT:

Background

A first therapeutic target of somatic genome editing (SGE) is sickle cell disease (SCD), the most commonly inherited blood disorders, affecting more than 100,000 individuals in the United States. Advancement of SGE is contingent on patient participation in first in human clinical trials. However, seriously ill patients may be vulnerable to overestimating the benefits of early phase studies while underestimating the risks. Therefore, ensuring potential clinical trial participants are fully informed prior to participating in a SGE clinical trial is critical. Methods: We conducted a mixed-methods study of adults with SCD as well as parents and physicians of individuals with SCD. Participants were asked to complete a genetic literacy survey, watch an educational video

SUBMITTER: Desine S 

PROVIDER: S-EPMC7710006 | biostudies-literature | 2020 Oct-Dec

REPOSITORIES: biostudies-literature