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http://dx.doi.org/10.1620/tjem.2025.J103 | DOI Listing |
JCI Insight
September 2025
Edinburgh Medical School: Biomedical Sciences & Euan MacDonald Centre for M, University of Edinburgh, Edinburgh, United Kingdom.
Spinal muscular atrophy (SMA) is a neuromuscular disease caused by low levels of SMN protein. Several therapeutic approaches boosting SMN are approved for human patients, delivering remarkable improvements in lifespan and symptoms. However, emerging phenotypes, including neurodevelopmental comorbidities, are being reported in some treated SMA patients, indicative of alterations in brain development.
View Article and Find Full Text PDFJCI Insight
September 2025
Division of Nephrology, Boston University Chobanian & Avedisian School of Medicine, Boston, United States of America.
Background: Active vitamin D metabolites, including 25-hydroxyvitamin D (25D) and 1,25-dihydroxyvitamin D (1,25D), have potent immunomodulatory effects that attenuate acute kidney injury (AKI) in animal models.
Methods: We conducted a phase 2, randomized, double-blind, multiple-dose, 3-arm clinical trial comparing oral calcifediol (25D), calcitriol (1,25D), and placebo among 150 critically ill adult patients at high-risk of moderate-to-severe AKI. The primary endpoint was a hierarchical composite of death, kidney replacement therapy (KRT), and kidney injury (baseline-adjusted mean change in serum creatinine), each assessed within 7 days following enrollment using a rank-based procedure.
JCI Insight
September 2025
Department of Pharmacology, University of Michigan, Ann Arbor, United States of America.
Cardiac hypertrophy is a common adaptation to cardiovascular stress and often a prelude to heart failure. We examined how S-palmitoylation of the small GTPase, Ras-related C3 botulinum toxin substrate 1 (Rac1), impacts cardiomyocyte stress signaling. Mutation of the cysteine-178 palmitoylation site impaired activation of Rac1 when overexpressed in cardiomyocytes.
View Article and Find Full Text PDFMol Pharm
September 2025
Department of Patho-Functional Bioanalysis, Graduate School of Pharmaceutical Sciences, Kyoto University, 46-29 Yoshida Shimoadachi-cho, Sakyo-Ku, Kyoto 606-8501, Japan.
Fibroblast activation protein (FAP) is an attractive biomarker for tumor-targeting radioligands. While [Ga]Ga-FAPI-46 is a promising FAP-targeting radioligand for cancer diagnosis, clinical application of [Lu]Lu-FAPI-46 for targeted radionuclide therapy is limited due to its insufficient tumor retention. Albumin binder (ALB) including 4-(-iodophenyl)butyric acid is widely utilized to improve tumor accumulation of radioligands.
View Article and Find Full Text PDFClin J Am Soc Nephrol
September 2025
Kidney Division, Peking University First Hospital, Peking University Institute of Nephrology; Key Laboratory of Kidney Disease, Ministry of Health of China; Key Laboratory of Chronic Kidney Disease Prevention and Treatment (Peking University), Ministry of Education, China.
Background: The Therapeutic Effects of Steroids in IgA Nephropathy Global (TESTING) trial demonstrated that glucocorticoid therapy reduced proteinuria and improved kidney outcomes in patients with Immunoglobulin A Nephropathy (IgAN). Galactose-deficient IgA1 (Gd-IgA1) plays a central role in IgAN pathogenesis by promoting immune complex formation. However, the effects of glucocorticoid on pathogenic IgA levels remain unclear.
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