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| http://dx.doi.org/10.1161/CIRCULATIONAHA.120.051940 | DOI Listing |
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Rational engineering of minimally immunogenic nucleases for gene therapy.
Nat Commun
January 2025
Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.
Genome editing using CRISPR-Cas systems is a promising avenue for the treatment of genetic diseases. However, cellular and humoral immunogenicity of genome editing tools, which originate from bacteria, complicates their clinical use. Here we report reduced immunogenicity (Red)(i)-variants of two clinically relevant nucleases, SaCas9 and AsCas12a.
View Article and Find Full Text PDFAnalysis of changes in PCSK9 and TNF-α concentrations in response to physical exercises using virtual reality in patients in the fifth stage of chronic kidney disease undergoing hemodialysis.
BMC Nephrol
January 2025
Department of Medical Rehabilitation and Clinical Physiotherapy, Pomeranian Medical University, Żołnierska 54, Szczecin, 71-210, Poland.
Article Synopsis
- The study investigates the effectiveness of virtual reality (VR) integrated exercise during hemodialysis for patients with stage 5 Chronic Kidney Disease (CKD).
- The research utilized the NefroVR system, allowing patients to perform regular physical training with VR technology.
- Results showed a significant reduction in harmful proteins PCSK9 and TNF-α after three months, indicating potential health benefits from this innovative exercise method.
PCSK9 in T-cell function and the immune response.
Biomark Res
December 2024
Department of Hematology, Shandong Provincial Hospital Affiliated to Shandong First Medical University, Shandong, 250021, China.
Proprotein convertase subtilisin/kexin type 9 (PCSK9) was first reported in 2003 and confirmed to be strongly associated with familial hypercholesterolemia. Small-molecule inhibitors targeting PCSK9 provide an effective and safe method for managing hypercholesterolemia and reducing the cardiovascular risk. In recent years, increasing evidence has indicated other important roles for PCSK9 in inflammation, tumors, and even immune regulation.
View Article and Find Full Text PDFFamilial hypercholesterolemia in Chinese children and adolescents: a multicenter study.
Lipids Health Dis
December 2024
Department of Endocrinology, Children's Hospital of Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, Zhejiang, 310052, China.
Background: Familial hypercholesterolemia (FH) is an inherited disorder mainly marked by increased low-density lipoprotein cholesterol (LDL-C) concentrations and a heightened risk of early-onset arteriosclerotic cardiovascular disease (ASCVD). This study seeks to characterize the genetic spectrum and genotype‒phenotype correlations of FH in Chinese pediatric individuals.
Methods: Data were gathered from individuals diagnosed with FH either clinically or genetically at multiple hospitals across mainland China from January 2016 to June 2024.
PCSK9 inhibitor alleviates experimental pulmonary fibrosis-induced pulmonary hypertension via attenuating epithelial-mesenchymal transition by suppressing Wnt/β-catenin signaling and .
Front Med (Lausanne)
December 2024
Medical College of Soochow University, Suzhou, Jiangsu, China.
Background: The co-occurrence of pulmonary hypertension (PH) in patients with pulmonary fibrosis (PF) is linked to a more unfavorable prognosis and increased mortality compared to PF cases without PH. Early intervention and comprehensive management are pivotal for improving survival outcomes. Proprotein convertase subtilisin/kexin type 9 (PCSK9) is a protein essential in cholesterol metabolism.
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