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http://dx.doi.org/10.1001/jamaoncol.2023.0239 | DOI Listing |
Exp Mol Med
January 2025
Department of Physiology, Yonsei University College of Medicine, 50-1 Yonsei-ro, Seodaemun-gu, Seoul, 03722, Korea.
Hemophilia A (HA) is caused by mutations in coagulation factor VIII (FVIII). Genome editing in conjunction with patient-derived induced pluripotent stem cells (iPSCs) is a promising cell therapy strategy, as it replaces dysfunctional proteins resulting from genetic mutations with normal proteins. However, the low expression level and short half-life of FVIII still remain significant limiting factors in the efficacy of these approaches in HA.
View Article and Find Full Text PDFJ Mol Histol
January 2025
Department of Histology, Faculty of Medicine, Assiut University, Assiut, Egypt.
Autism spectrum disorder (ASD) is a group of severe neurodevelopmental disorders. This study aimed to elucidate the potential ameliorating effect of postnatal administration of MSCs-derived Exo in a rat model of ASD. Male pups were divided into control (Cont), (VPA); pups of pregnant rats injected with VPA subcutaneously (S.
View Article and Find Full Text PDFCancer Immunol Immunother
January 2025
Department of Oncology, Lianyungang Clinical College of Nanjing Medical University/The First People's Hospital of Lianyungang, The Affiliated Lianyungang Hospital of Xuzhou Medical University, Lianyungang, 222002, China.
Background: Due to its strong immunogenicity and tumor specificity, neoplastic antigen has emerged as an immunotherapy target with wide therapeutic prospect and clinical application value. Anti-programmed death-1 (PD-1) antibodies reinvigorate T cell-mediated antitumor immunity. So, we conducted single-arm trial to assess the safety and efficacy of PD-1 blockade(Camrelizumab)-activated neoantigen specific cellular therapy (aNASCT) on advanced relapsed non-small lung cancer(NSCLC)(ClinicalTrials.
View Article and Find Full Text PDFActa Biomater
December 2024
The Second Affiliated Hospital, Medical Science and Technology Innovation Center, Shandong First Medical University & Shandong Academy of Medical Sciences, Jinan, Shandong 250117, China. Electronic address:
Toll-like receptor (TLR) 7/8 agonists have shown significant potential in tumor immunotherapy. However, the limited pharmacokinetic properties and systemic toxicity resulting from off-target effects limits their biomedical applications. We here report the polyphenol-mediated assembly of resiquimod (R848, a TLR7/8 agonist) nanoparticles (RTP NPs) to achieve tumor-selective immunotherapy while avoiding systemic adverse effects.
View Article and Find Full Text PDFMol Pharm
January 2025
Drug Product Development-Steriles, Medicine Development and Supply, GSK, 1250 S. Collegeville Road, Collegeville, Pennsylvania 19426, United States.
Therapeutic monoclonal antibody (mAb) drug products are increasingly used to treat both chronic and acute diseases. These mAb drug products are often developed for subcutaneous (SC) injection to simplify dosing compared with intravenous (IV) infusion. For SC injection, the mAb liquid drug product is typically filled in a vial for use with a syringe or in a prefilled syringe, which can then be assembled into a safety syringe device or an autoinjector for direct administration.
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