Differences in the autoantibody phenotypes and long-term outcomes between juvenile- and adult-onset systemic sclerosis.

[Objective] To investigate differences in autoantibodies, clinical features, and long-term outcomes between juvenile- and adult-onset systemic sclerosis (SSc). [Methods] Autoantibodies and survival rates over a maximum of 20 years were retrospectively analyzed in 504 Japanese patients with SSc (juvenile-onset SSc, n=17; adult-onset SSc, n=487) using data from Kyoto University Registry. [Results] The autoantibodies observed were anti-topoisomerase-I (71% vs.

Validity and reliability of the Japanese version of the ACE tool for assessing evidence-based medicine competencies in medical practitioners and students: An evaluation in an online setting.

Introduction Evidence-based medicine (EBM) competency is crucial for healthcare professionals; however, validated tools to assess EBM skills in Japanese are scarce. This study aimed to develop and validate a Japanese version of the Assessing Competency in EBM (ACE) tool. Methods We translated the ACE tool into Japanese, following international standards, and distributed it online to 99 healthcare professionals and students.

Kidney Injury in Patient with Non-small-cell Lung Cancer Receiving Tepotinib.

We herein report a 68-year-old man with advanced non-small-cell lung cancer treated with tepotinib who showed marked general edema, hypoalbuminemia, and an elevated serum creatinine level. Although tepotinib-induced kidney injury due to creatinine transporter inhibition has been reported, renal biopsy findings suggested tubulointerstitial injury due to decreased renal blood flow, likely secondary to refractory fluid retention. This case highlights the potential for true kidney injury during tepotinib therapy and underscores the importance of careful monitoring and management of adverse renal effects.

Optimization of HITI-Mediated Gene Insertion for Rhodopsin and Peripherin-2 in Mouse Rod Photoreceptors: Targeting Dominant Retinitis Pigmentosa.

Purpose: Among the genome-editing methods for repairing disease-causing mutations resulting in autosomal dominant retinitis pigmentosa, homology-independent targeted integration (HITI)-mediated gene insertion of the normal form of the causative gene is useful because it allows the development of mutation-agnostic therapeutic products. In this study, we aimed for the rapid optimization and validation of HITI-treatment gene constructs of this approach in developing HITI-treatment constructs for various causative target genes in mouse models of retinal degeneration.

Methods: We constructed the Cas9-driven HITI gene cassettes in plasmid vectors to treat the mouse Rho gene.