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A novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary results.


ABSTRACT:

Introduction

Despite the progress in gene editing platforms like CRISPR/Cas9 with the potential to transform the standard of care for haemophilia, the language used to explain and discuss gene editing is not aligned across the haemophilia community. Here, we present the objective and rationale for developing a clear, consistent, and globally aligned gene editing lexicon to address these communication gaps.

Methods

Effectively communicating complex gene editing concepts requires a clear and consistent vocabulary. Through collaboration with a diversity of haemophilia stakeholders, our main goal is to develop an accurate, informative lexicon which avoids overpromising or highly technical terminology. Using an innovative process, representatives from several patient and scientif

SUBMITTER: Hermans C 

PROVIDER: S-EPMC11659488 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature