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Long-term correction of hemophilia A via integration of a functionally enhanced FVIII gene into the AAVS1 locus by nickase in patient-derived iPSCs.
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 PDFSynthesis of Schiff bases based on Chitosan, thermal stability and evaluation of antimicrobial and antitumor activities.
Sci Rep
January 2025
Department of Chemistry, Faculty of Science, Al-Azhar University, Nasr City, 11884, Cairo, Egypt.
A Schiff base of Chitosan was prepared by condensing of the Chitosan (CS) with six aromatic aldehydes and confirmed by FT-IR, NMR, XRD, TGA, and DSC. XRD results showed the disappeared of peaks at 2θ = 10° for CS and appeared one peaks at around 2θ of 23° for Schiff bases, while TGA was demonstrated that the thermal stability of CS has improved after the modification with the corresponding aldehyde. Also, DSC shows endothermal peak of CS at 100 °C due to the loss of water and second thermal event related to the decomposition of amine units with an exothermic peak at 295 °C, while Schiff bases shows endothermal peak around 70-95 °C which is related to the loss of water for all samples and the second exothermic peak around 260-280 °C is related to the decomposition of the amine group in the polymer units.
View Article and Find Full Text PDFMycosynthesis of chitosan-selenium nanocomposite and its activity as an insecticide against the cotton leafworm Spodoptera littoralis.
Sci Rep
January 2025
Botany and Microbiology Department, Faculty of Science, Zagazig University, Zagazig, Egypt.
The cotton leafworm, Spodoptra littoralis, causes great damage to cotton crops. A new, safer method than insecticide is necessary for its control. Selenium nanoparticles (SeNPs) are metalloid nanomaterial, with extensive biological activities.
View Article and Find Full Text PDFNano-chitosan-coated, green-synthesized selenium nanoparticles as a novel antifungal agent against Sclerotinia sclerotiorum: in vitro study.
Sci Rep
January 2025
Biophysics Department, Faculty of Science, Cairo University, Giza, Egypt.
Chemical fungicides have been used to control fungal diseases like Sclerotinia sclerotiorum. These fungicides must be restricted because of their toxicity and the development of resistance strains. Therefore, utilizing natural nanoscale materials in agricultural production is a potential alternative.
View Article and Find Full Text PDFProduction of Recombinant Adeno-Associated Virus Through High-Cell-Density Transfection of HEK293 Cells Based on Fed-Perfusion Culture.
Hum Gene Ther
January 2025
School of Bioengineering, East China University of Science and Technology, Shanghai, China.
Adeno-associated virus (AAV)-associated gene therapy has been increasingly promising, in light of the drugs progressed to clinical trials or approved for medications internationally. Therefore, scalable and efficient production of recombinant AAV is pivotal for advancing gene therapy. Traditional methods, such as the triple-plasmid transfection of human embryonic kidney 293 cells in suspension culture, have been widely employed but often hampered by low unit yield.
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