Therapeutic effect of TTR siRNA on hereditary transthyretin amyloidosis (ATTRv) nephropathy.

IF 5.2 2区医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY

Amyloid-Journal of Protein Folding Disorders Pub Date : 2025-02-23 DOI:10.1080/13506129.2025.2470369

Takahiro Ajiki, Tatsuhiko Ozono, Fusako Sera, Hiroaki Yonishi, Jun Matsuda, César Aguirre, Keita Kakuda, Yumiko Yasuhara, Yoshitaka Isaka, Yasushi Sakata, Kensuke Ikenaka, Hideki Mochizuki

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引用次数: 0

Abstract

Background: Hereditary transthyretin amyloidosis (ATTRv) is a fatal disease that affects multiple organs. Up to 30% of patients with ATTRv also experience renal complications, including proteinuria and a decline in eGFR. Recently, new treatments for ATTRv, a tetramer stabiliser and transthyretin small interfering RNA (TTR siRNA) therapeutics, have emerged. However, the effectiveness of these new treatments on renal complications in ATTRv remains unknown.

Methods: We retrospectively collected clinical data from ATTRv patients and analysed the relationship between the initial renal complications and age. We also examined whether the new treatments affected the clinical course of renal symptoms, using eGFR changes or longitudinal data on urine protein/albumin creatinine ratio.

Results: A total of 16 patients' data were collected. Regarding their initial renal complications, we found that patients with proteinuria had an earlier age at onset than those with a decline in eGFR. Notably, longitudinal data showed that TTR siRNA therapeutics reduced proteinuria and increased serum protein, while none of the new treatments could demonstrate a significant improvement in the slope of eGFR decline.

Conclusions: We demonstrated that TTR siRNA therapeutics represent potential candidates for ATTRv nephropathy, despite the fact that their use has been limited to neurological symptoms to date.

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来源期刊

Amyloid-Journal of Protein Folding Disorders 生物-生化与分子生物学

CiteScore

10.60

自引率

10.90%

发文量

审稿时长

6-12 weeks

期刊介绍： Amyloid: the Journal of Protein Folding Disorders is dedicated to the study of all aspects of the protein groups and associated disorders that are classified as the amyloidoses as well as other disorders associated with abnormal protein folding. The journals major focus points are: etiology, pathogenesis, histopathology, chemical structure, nature of fibrillogenesis; whilst also publishing papers on the basic and chemical genetic aspects of many of these disorders. Amyloid is recognised as one of the leading publications on amyloid protein classifications and the associated disorders, as well as clinical studies on all aspects of amyloid related neurodegenerative diseases and major clinical studies on inherited amyloidosis, especially those related to transthyretin. The Journal also publishes book reviews, meeting reports, editorials, thesis abstracts, review articles and symposia in the various areas listed above.