Stem Cell-Based Therapeutic Approaches in Genetic Diseases.

Stem cells, which can self-renew and differentiate into different cell types, have become the keystone of regenerative medicine due to these properties. With the achievement of superior clinical results in the therapeutic approaches of different diseases, the applications of these cells in the treatment of genetic diseases have also come to the fore. Foremost, conventional approaches of stem cells to genetic diseases are the first approaches in this manner, and they have brought safety issues due to immune reactions caused by allogeneic transplantation. To eliminate these safety issues and phenotypic abnormalities caused by genetic defects, firstly, basic genetic engineering practices such as vectors or RNA modulators were combined with stem cell-based therapeutic approaches. However, due to challenges such as immune reactions and inability to target cells effectively in these applications, advanced molecular methods have been adopted in ZFN, TALEN, and CRISPR/Cas genome editing nucleases, which allow modular designs in stem cell-based genetic diseases' therapeutic approaches. Current studies in genetic diseases are in the direction of creating permanent treatment regimens by genomic manipulation of stem cells with differentiation potential through genome editing tools. In this chapter, the stem cell-based therapeutic approaches of various vital genetic diseases were addressed wide range from conventional applications to genome editing tools.

Biogenic synthesis, molecular docking, biomedical and environmental applications of multifunctional CuO nanoparticles mediated Phragmites australis.

The demand for metal nanoparticles is increasing with the widening application areas while causing environmental impact including pollution, toxic byproduct generation and depletion of natural resources. Incorporating natural materials in nanoparticle synthesis can contribute toward environmental sustainability. This paper is concerned with the biogenic synthesis of copper oxide nanoparticles (CuONPs) mediated by the plant species Phragmites australis. UV-vis, FT-IR, TEM and SEM studies were used to characterize the obtained CuONPs. The synthesized nanoparticles' antibacterial efficacy against Escherichia coli and Staphylococcus aureus was assessed. The CuONPs' reducing power, total phenolic component content, and flavonoid content were all calculated. Additionally, the dye removal abilities of copper oxide nanoparticles using Brilliant Blue R-250 were studied. The CuONP synthesis was assessed morphological by change of color and in the UV-vis analysis by the SPR band around 320 and 360 nm. FT-IR was used to monitor the functional groups present in the synthesized CuONPs. The obtained CuONPs were spherical and between 70 and 142 nm in size, according to the SEM data and TEM analyses were in accordance with SEM results. Using disk diffusion, the CuONPs demonstrated substantial antibacterial efficacy against S. aureus and E. coli, with inhibition zones of 18.5 ± 0.8 and 12.7 ± 0.6 mm, respectively. The MBC and MIC values were 62.5 µg/mL against S. aureus and 125 µg/mL against E. coli. The antioxidant abilities of P. australis and CuONPs were also confirmed. The CuONP solution's total phenolic substance content was 9.44 µg of pyrocathecol equivalent per milligram of nanoparticle, and its total flavonoid content was 16.24 µg of catechin equivalent per milligram of nanoparticle. Additionally, the synthesized CuONPs were found to be well effective on industrial dye removal by demonstrating high decolorization of 98 %. Also, the antibacterial activity of CuONPs was investigated through the interactions with S. aureus FtsZ, dihydropteroate synthase and thymidylate kinase. In silico molecular docking analysis was applied in the confirmation of the binding sites and interactions of active sites. CuONP showed -9.067, -8,048, and -7.349 kcal/mol of binding energies in molecular docking analysis of FtsZ, dihydropteroate synthase and thymidylate kinase proteins respectively. The results of this study suggested the antimicrobial, antioxidant and decolorative effect of synthesized CuONPs that can be apply in multiple areas of R&D and industry.