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Precise correction of G6PD Viangchan mutation in iPSCs by prime editing strategy
Chaiwat Netsawang1, Malinee Tongbaen1, Natee Jearawiriyapaisarn2
1Programme in Clinical Hematology Sciences, Department of Clinical Microscopy, Faculty of Allied Health Sciences, Chulalongkorn University, Bangkok, Thailand.
Scientific Reports
|August 18, 2025
Summary
Prime editing effectively corrects the prevalent G6PD Viangchan mutation in cell lines. This gene editing tool shows high precision and minimal by-products, offering a promising therapeutic strategy for glucose-6-phosphate dehydrogenase deficiency.
Area of Science:
- Genetics
- Molecular Biology
- Biotechnology
Background:
- Glucose-6-phosphate dehydrogenase (G6PD) deficiency poses a risk of hemolytic crises due to oxidative stress.
- No definitive cure currently exists for G6PD deficiency.
- G6PD Viangchan is the most common variant, particularly in Southeast Asia.
Purpose of the Study:
- To evaluate prime editing's efficacy in correcting the G6PD Viangchan mutation.
- To assess prime editing in both HEK293T cells and patient-derived induced pluripotent stem cells (iPSCs).
Main Methods:
- Utilized prime editing technology to target the G6PD Viangchan mutation (c.871 G>A).
- Applied optimized prime editing modalities in HEK293T cells and G6PD-deficient iPSCs.
Main Results:
- Achieved over 25% correction efficiency in HEK293T cells.
- Demonstrated approximately 5% allele correction in iPSCs.
- Prime editing exhibited high precision with minimal by-products and undetectable off-target effects.
Conclusions:
- Prime editing shows significant potential for correcting the G6PD Viangchan mutation.
- This approach could lead to future therapeutic strategies for G6PD deficiency.
- Facilitates the creation of isogenic cell lines for drug discovery and pathogenesis research.
