Harnessing Gene Correction for Endemic Disease: A Review of CRISPR-Cas9 Potential in Treating Genetic Diseases in Africa

Abstract

Clustered regularly interspaced short palindromic repeats (CRISPR) alongside Cas9 has been a promising innovative genomic editing technology with potential to treat genetic diseases. It offers scientists the chance to edit DNA structures and change gene function. This review synthesises pre-clinical and early-phase data on its application against Africa’s highest-burden inherited disorders sickle-cell disease, β-thalassaemia, glucose-6-phosphate dehydrogenase (G6PD) deficiency and ornithine transcarbamylase (OTC) deficiency, cystic fibrosis, Duchenne muscular dystrophy and HIV-1 infection many of which are endemic and carry African-specific mutations. Using a structure review approach, we analysed > 80 peer-reviewed studies published 2015-2025 that employed African donor cells or relevant animal models. Key findings include ≥ 70 % on-target correction of prevalent variants (G6PD A− variant, CFTR A559T, BCL11A enhancer), functional rescue of enzyme activity or viral resistance, and absence of high-level off-target events when high-fidelity nucleases are used. The convergence of falling reagent costs, continent-specific mutation atlases and newly commissioned GMP facilities provides a realistic pathway for First-in-Human trials within 5–7 years. Implications include the potential to leapfrog from lifelong palliative care to one-shot cures, provided that Africa builds scientific infrastructure, trains local genome engineers and enacts harmonised regulatory frameworks that are both stringent and context-sensitive.