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Strong protective effect of the APOL1 p.N264K variant against G2-associated FSGS and kidney disease

Gupta, Y.; Friedman, D. J.; McNulty, M.; Khan, A.; Lane, B.; Wang, C.; Ke, J.; Jin, G.; Wooden, B.; Knob, A. L.; Lim, T. Y.; Appel, G. B.; Huggins, K.; Liu, L.; Mitrotti, A.; Stangl, M. C.; Bomback, A.; Westland, R.; Bodria, M.; Marasa, M.; Shang, N.; Cohen, D. J.; Crew, R. J.; Morello, W.; Canetta, P.; Radhakrishnan, J.; Martino, J.; Liu, Q.; Chung, W. K.; Espinoza, A.; Luo, Y.; Wei, W.-Q.; Feng, Q.; Weng, C.; Fang, Y.; Kullo, I. J.; Naderian, M.; Limdi, N.; Irvin, M. R.; Tiwari, H.; Mohan, S.; Rao, M.; Dube, G.; Chaudhary, N. S.; Gutierrez, O. M.; Judd, S. E.; Cushman, M.; Lange, L. A.; Lang

2023-08-04 genetic and genomic medicine
10.1101/2023.08.02.23293554 medRxiv
Show abstract

Black Americans have a significantly higher risk of developing chronic kidney disease (CKD), especially focal segmental glomerulosclerosis (FSGS), than European Americans. Two coding variants (G1 and G2) in the APOL1 gene play a major role in this disparity. While 13% of Black Americans carry the high-risk recessive genotypes, only a fraction of these individuals develops FSGS or kidney failure, indicating the involvement of additional disease modifiers. Here, we show that the presence of the APOL1 p.N264K missense variant, when co-inherited with the G2 APOL1 risk allele, substantially reduces the penetrance of the G1G2 and G2G2 high-risk genotypes by rendering these genotypes low-risk. These results align with prior functional evidence showing that the p.N264K variant reduces the toxicity of the APOL1 high-risk alleles. These findings have important implications for our understanding of the mechanisms of APOL1-associated nephropathy, as well as for the clinical management of individuals with high-risk genotypes that include the G2 allele.

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