Throwing off the keratin chains: a potential therapy for hereditary podocytopathy

Kálmán Tory

doi:10.1016/j.kint.2024.01.029

Throwing off the keratin chains: a potential therapy for hereditary podocytopathy

Kidney Int. 2024 Apr;105(4):663-665. doi: 10.1016/j.kint.2024.01.029.

Author

Kálmán Tory¹

Affiliation

¹ Pediatric Center, Semmelweis University, Budapest, Hungary; Hungarian Academy of Sciences - Semmelwies University (MTA-SE) Lendület Nephrogenetic Laboratory, Semmelweis University, Budapest, Hungary. Electronic address: [email protected].

PMID: 38519231
DOI: 10.1016/j.kint.2024.01.029

Abstract

In the current issue, Kuzmuk et al. offer a therapeutic option for patients with NPHS2 R138Q-associated nephrotic syndrome. For the first time in hereditary podocytopathies, this is offered by restoring the membrane targeting of a pathogenic protein. The idea that it is enough to liberate podocin from the trap of keratin 8, a key member of endoplasmic-reticulum-associated protein degradation complex, was brilliantly recognized based on former results obtained in cystic fibrosis.

MeSH terms

Humans
Keratins* / therapeutic use
Mutation
Nephrotic Syndrome* / drug therapy
Nephrotic Syndrome* / genetics

Substances

Keratins