APOL1-associated kidney disease: recent advances from genetic mechanisms to targeted therapy

doi:10.3877/cma.j.issn.2095-3216.2026.01.006

Abstract

Abstract:

Apolipoprotein L1-associated kidney disease is a renal disorder mediated by risk variants in the apolipoprotein L1 (APOL1) gene, predominantly manifesting as focal segmental glomerulosclerosis (FSGS). Currently, there is no specific therapy, and the efficacy of existing non-specific treatment regimens remains suboptimal. With the deepening understanding of the pathogenic mechanisms underlying APOL1 G1/G2 risk variants and the recently reported p. T272I variant, targeted therapeutic agents, such as small-molecule inhibitors, antisense oligonucleotides, and Janus kinase inhibitors, have successively entered clinical investigation. This article reviews the latest advances in APOL1-associated kidney disease, spanning from genetic mechanisms to targeted therapeutics.

Key words: Apolipoprotein L1, Chronic kidney disease, Focal segmental glomerulosclerosis, Gene variants, Targeted treatment

Zhiyao He, Jia Chen, Kehong Chen. APOL1-associated kidney disease: recent advances from genetic mechanisms to targeted therapy[J]. Chinese Journal of Kidney Disease Investigation(Electronic Edition), 2026, 15(01): 34-38.

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