Published December 27, 2025 | Version V1

CRISPR-Cas9 Genome Editing: Clinical Promise, Global Inequity, and Governance Challenges

Authors/Creators

  • 1. Independent Researcher

Description

CRISPR-Cas9 can cure sickle cell disease, yet 80% of patients with this condition—most of whom live in sub-Saharan Africa—will never access this therapy. Therapeutic success without equitable access and robust governance represents a new form of health inequity rather than medical triumph. This review examines the progression of genome editing from laboratory tool to approved therapy, analyzes why current delivery and manufacturing models limit global access, and evaluates governance frameworks required before germline editing becomes technically inevitable. Clinical trials report notable efficacy: exagamglogene autotemcel achieved 94% vaso-occlusive crisis-free survival and 89% transfusion independence. However, significant barriers persist: off-target effects, delivery limitations, manufacturing costs exceeding $2.2 million per treatment, and fragmented international governance. The path forward requires reconceptualizing who benefits from biomedical innovation—not as an afterthought, but as a fundamental design principle. Technical progress alone is insufficient; the defining challenge is ensuring genome editing serves broadly shared human values rather than only those with economic privilege.

Files

Kiani_CRISPR_Cas9_Preprint_Zenodo-2.pdf

Files (278.9 kB)

Name Size
md5:32bc4b2a394d58fcc1b3005d5ddb108d
278.9 kB Preview Download