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Gene Therapy in Oral Tissue Regeneration
Published in Vincenzo Guarino, Marco Antonio Alvarez-Pérez, Current Advances in Oral and Craniofacial Tissue Engineering, 2020
Fernando Suaste, Patricia González-Alva, Alejandro Luis, Osmar Alejandro
Transfection is, therefore, a method designed for the introduction of genetic elements (coding and non-coding DNA sequences) that control the expression of a gene, either by increasing its expression or suppressing it. In the former, transfection can be used in gene therapy for the treatment of diseases or induce changes in cell reprogramming by introducing transcription factors into mesenchymal cells (cell renewal). Moreover, transfection can be used to reduce the expression of a gene, through RNAi, or by performing genetic editing to correct errors in the sequence or eliminating them through the CRISPR/Cas system (Glorioso and Lemoine 2017; Anguela and High 2018).
Application of Bioresponsive Polymers in Gene Delivery
Published in Deepa H. Patel, Bioresponsive Polymers, 2020
Tamgue Serges William, Drashti Pathak, Deepa H. Patel
Multi Stimuli-Responsive Systems: This approach is based on the combination of more than one stimulus for the delivery of nucleic acids, with the purpose to overcome intracellular multistep barriers that limit high levels of gene expression. It involves the modification of polymer with specific groups or moieties in the way that it can sense different stimuli at the same time, which can be pH, temperature, ROS, or another one. This approach could be the best one to achieve high transfection efficiencies similar to those of viruses.
Molecular Radiation Biology
Published in Kedar N. Prasad, Handbook of RADIOBIOLOGY, 2020
The availability of plasmid and retroviral vectors carrying specific oncogenes has allowed us to insert exogenous genes into any cell type. The process of gene insertion is often referred to as transfection. The transfection of cells with specific genes can be accomplished by several methods, including electroporation and calcium-phosphate-Co-DNA precipitation techniques. The role of cellular oncogenes or genes in radiosensitivity of mammalian cells can be studied by generating stable transfectants that express high levels of genes under investigation.
Pitfalls in patenting academic CAR-T cells therapy
Published in Expert Opinion on Therapeutic Patents, 2023
Cătălin Constantinescu, Roxana Constantinescu, Jon Thor Bergthorsson, Victor Greiff, Alina Tanase, Anca Colita, Diana Gulei, Ciprian Tomuleasa
The genetic sequences that contain the code for these polypeptides may also provide protection for the CARs and the parts that make them up. Given that the CAR is only expressed in the patient’s cells, it will be difficult to demonstrate infringement of a polypeptide claim. However, before transfection, the nucleic acid can be synthesized and purified in bulk, much like any other small molecule or biotech item. Thus, a patentee can prove infringement for that polynucleotide in general. In addition, patent protection should be sought for newly developed functional subunits of a CAR, such as targeting moieties and co-stimulatory domains. For example, it was recently reported that a peptide fragment of scorpion chlorotoxin can be used to effectively target glioblastoma cells [34]. This targeting mechanism can receive large protection from drafting the claims in a manner that ensures viable association between peptide fragments and other functional fields of a CAR.
Recent progress in small molecule TBK1 inhibitors: a patent review (2015– 2020)
Published in Expert Opinion on Therapeutic Patents, 2021
Douglas W. Thomson, Giovanna Bergamini
As well as direct applications in the treatment of disease, TBK1 inhibitors also find uses in more fundamental research. In this respect, Rosa Karl disclosed the use of TBK1 inhibitors for improving the efficiency of transfection of cells with non-viral nucleic acids [39,40]. Transfection of cells enables scientists to control the expression of genes and has become a key technology to explore biology and has therapeutic applications. However, the innate immune system can restrict the success of the transfection as it is activated by cytosolic nucleic acids. Karl showed that transfection efficiency is improved by treating the cells with a TBK1 inhibitor during transfection. A further improvement in transfection is seen with co-treatment of a TBK1 inhibitor and nucleic-acid-detecting toll like receptor inhibitor. Based on the same principle Precision Biosciences claimed methods for reducing DNA-induced cytotoxicity and enhancing gene editing in primary cells [41]. Precision scientists claimed the use of inhibitors in the cGAS-STING-TBK1 signaling pathway to increase transfection efficiency. However, they focused on the transfection of primary cells for preclinical research and therapeutics, for example the use of modified human T cells for the treatment of cancer.
Effects of human cyclooxygenase-2 gene silencing on synovial cells of rheumatoid arthritis mediated by lentivirus
Published in Artificial Cells, Nanomedicine, and Biotechnology, 2018
Ping Leng, Dawei Li, Yi Sun, Yingzhen Wang, Haining Zhang
After inhibition of COX-2, the inflammation factors such as PGE2, VEGF, TNF-α could be down-regulated. The reduction of IL-1β may be ascribed to complex signalling pathway, and the inhibition efficiency was similar with NS398 in 500 nmol/l concentration on synovial cells. In a study about retrovirus infection, transducing efficiency at 60–70% was obtained in primary human synovial fibroblasts under the conditions of MOI ranged between 120 and 12 T.U./cell, when 2000 or 20,000 cell/cm2 were seeded. In the current research, transfection efficiency at 70% was achieved when the MOI was 70–100, while obvious toxicity on the cells occurred when the MOI was higher than 100. Higher transfection efficiency and inhibition ratio appeared 5–10 days later after transfection. Therefore, COX-2 siRNA transfection mediated by lentivirus is a theoretically effective way to control the inflammatory reaction in synovium of RA patients. Since multiple target sequences according to the COX-2 mRNA can be designed for gene silencing, more efficient COX-2 shRNA can be screened for further clinical trial.