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Adenine base editor corrected ADPKD point mutations in hiPSCs and kidney organoids.
Adv Biotechnol (Singap)
June 2024
MOE Key Laboratory of Gene Function and Regulation, State Key Laboratory of Biocontrol, School of Life Sciences, Sun Yat-Sen University, Guangzhou, Guangdong, 510275, China.
Autosomal dominant polycystic kidney disease (ADPKD) is a dominant genetic disorder caused primarily by mutations in the PKD1 gene, resulting in the formation of numerous cysts and eventually kidney failure. However, there are currently no gene therapy studies aimed at correcting PKD1 gene mutations. In this study, we identified two mutation sites associated with ADPKD, c.
View Article and Find Full Text PDFDecoding the genetic blueprint: regulation of key agricultural traits in sorghum.
Adv Biotechnol (Singap)
September 2024
School of Agriculture and Biotechnology, Sun Yat-sen University, Shenzhen, 518107, P. R. China.
Sorghum, the fifth most important crop globally, thrives in challenging environments such as arid, saline-alkaline, and infertile regions. This remarkable crop, one of the earliest crops domesticated by humans, offers high biomass and stress-specific properties that render it suitable for a variety of uses including food, feed, bioenergy, and biomaterials. What's truly exciting is the extensive phenotypic variation in sorghum, particularly in traits related to growth, development, and stress resistance.
View Article and Find Full Text PDFLowering LDL cholesterol by PCSK9 inhibition: a new era of gene silencing, RNA, and alternative therapies.
Naunyn Schmiedebergs Arch Pharmacol
January 2025
Institute of Pharmacy, Nirma University, Gujarat, 382481, India.
Proprotein convertase subtilisin/kexin type 9 (PCSK9) discovery has added a new paradigm to our understanding of cholesterol homeostasis and lipid metabolism. Since its discovery, PCSK9 inhibitors have become a widely investigated therapeutic class for lipid management in cardiovascular diseases and hypercholesterolemia. Scientists have explored different approaches for PCSK9 inhibition, such as monoclonal antibodies (mAbs), gene silencing and gene editing techniques, vaccines, mimetic peptides, and small molecules.
View Article and Find Full Text PDFSubstituent-Controlled Regiodivergent Rearrangement of Gramine Ammonium Ylide.
J Org Chem
January 2025
Key Laboratory of Tropical Biological Resources of Ministry of Education, School of Pharmaceutical Sciences, Hainan University, Haikou 570228, P.R. China.
The complicated mechanism makes the regiodivergent rearrangement of ammonium ylide seem to be out of reach. Herein, we reported a regiodivergent rearrangement of gramine ammonium ylide well controlled by the substituents. Density functional theory studies reveal that the ammonium ylide with a more steric hindrance substituent 2-diazo-2-arylacetate goes through a stepwise mechanism to yield both a kinetically and thermodynamically preferred [1,2]-rearrangement product.
View Article and Find Full Text PDFSilk Protein Gene Engineering and Its Applications: Recent Advances in Biomedicine Driven by Molecular Biotechnology.
Drug Des Devel Ther
January 2025
State Key Laboratory of Biobased Material and Green Papermaking, Qilu University of Technology, Shandong Academy of Sciences, Jinan, Shandong, 250353, People's Republic of China.
Silk protein, as a natural polymer material with unique structures and properties, exhibits tremendous potential in the biomedical field. Given the limited production and restricted properties of natural silk proteins, molecular biotechnology has been extensively applied in silk protein genetic engineering to produce novel silk proteins with specific properties. This review outlines the roles of major model organisms, such as silkworms and spiders, in silk protein production, and provides a detailed introduction to the applications of gene editing technologies (eg, CRISPR-Cas9), transgenic expression technologies, and synthetic biology techniques in silk protein genetic engineering.
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