Building a gene editing lexicon: a model for rare and inherited disorders

Rationale for developing a gene editing lexicon in hemophilia

The robust development of a gene editing lexicon for advanced treatments for hemophilia can serve as a template for other therapy areas in the gene therapy space, in which opportunities and barriers for patients are similar. Partnering with patient advocacy groups to support the adoption and dissemination of the lexicon ensures that patients remain the priority. Patient organizations could, in turn, influence how language is used in broader discussions of medical and scientific…