The use of genetically modified cells in modern biology and medicine
Genetically modified cells are an important research tool in biology and medicine. Their creation makes it possible to study the molecular mechanisms of various processes, model human diseases, develop new therapeutic approaches, and implement innovative biotechnologies. Modern genome-editing methods such as CRISPR/Cas9 provide high precision and efficiency of modifications, making genetically modified cells an integral part of cutting-edge science.
The process of creating genetically modified cells begins with selecting a target gene that needs to be modified. Various tools are used for editing, such as CRISPR/Cas9, TALENs, or ZFNs, which cleave DNA at a specific site. A donor template for correcting or replacing the target DNA region is then delivered into the cell [doi.org/10.1126/science.1225829]. One of the most promising directions is the use of CRISPR/Cas technologies. This mechanism is based on bacteria’s natural defense system against viruses, but in the hands of researchers it has become a powerful tool for editing cellular genomes. It enables a wide range of manipulations, from sequence changes to knockout of individual genes [doi.org/10.1038/s41580-019-0131-5].
A special tool for transgenesis is the Sleeping Beauty (SB) transposon (Fig. 1). It is a synthetically engineered system based on transposons from the Tc1/Mariner family and is currently considered one of the most promising tools for delivering and stably integrating genetic material into the genome of mammalian cells. Sleeping Beauty has become widely used due to its simple structure, high efficiency, and low risk of undesired genetic effects, such as integration into critical genes.
The process of creating genetically modified cells begins with selecting a target gene that needs to be modified. Various tools are used for editing, such as CRISPR/Cas9, TALENs, or ZFNs, which cleave DNA at a specific site. A donor template for correcting or replacing the target DNA region is then delivered into the cell [doi.org/10.1126/science.1225829]. One of the most promising directions is the use of CRISPR/Cas technologies. This mechanism is based on bacteria’s natural defense system against viruses, but in the hands of researchers it has become a powerful tool for editing cellular genomes. It enables a wide range of manipulations, from sequence changes to knockout of individual genes [doi.org/10.1038/s41580-019-0131-5].
A special tool for transgenesis is the Sleeping Beauty (SB) transposon (Fig. 1). It is a synthetically engineered system based on transposons from the Tc1/Mariner family and is currently considered one of the most promising tools for delivering and stably integrating genetic material into the genome of mammalian cells. Sleeping Beauty has become widely used due to its simple structure, high efficiency, and low risk of undesired genetic effects, such as integration into critical genes.
Figure 1. Sleeping Beauty transposon system. (A) Structure of the Sleeping Beauty transposon. The transposase gene (purple rectangle) is flanked by inverted repeats (IR, black arrows) that contain transposase-binding sites (white arrows). The transposase consists of an N-terminal DNA-binding domain, a nuclear localization signal (NLS), and a catalytic domain. (B) Genetic engineering Sleeping Beauty vector system. The transposase gene can be replaced with a gene of interest (yellow rectangle) positioned between the inverted repeats. The transposase gene is expressed from a separate plasmid carrying a promoter (black arrow) [doi.org/10.2174/156652306778520647].
SB-based technologies are widely used in gene therapy, cell engineering, and the development of personalized medicine. For example, SB helps in the genetic modification of T cells to generate CAR-T cells for cancer treatment, replacing the use of viral vectors, which may be potentially unsafe. In addition, the SB transposon is actively used to correct mutations underlying genetic diseases and to create disease models for research purposes [doi.org/10.2174/156652306778520647].
Applications of genetically modified cells
Disease modeling and investigation of molecular mechanisms
One of the most important applications of genetically modified cells is the creation of models of human diseases. For example, cells can be modified to mimic pathologies associated with specific genetic mutations, such as cystic fibrosis, Alzheimer’s disease, or cancer [doi.org/10.1016/j.tibtech.2011.04.009]. This makes it possible to study these diseases in vitro and to develop new treatment approaches.
Gene therapy
Genetically modified cells are also used in disease therapy. The most prominent example is CAR-T cancer therapy. This method involves the genetic modification of a patient’s T lymphocytes so that they express chimeric antigen receptors (CARs) capable of recognizing and destroying cancer cells. This approach has shown excellent results in the treatment of certain forms of leukemia and lymphoma [doi.org/10.1126/science.aar6711].
Biopharmaceuticals
Genetically modified cells are widely used in the production of biologic drugs. For example, engineered CHO cells are extensively used to produce antibodies and other protein therapeutics that are applied in the treatment of diseases such as cancer and rheumatoid arthritis.
Regenerative medicine and organoid generation
Another promising area is the use of genetically modified cells in regenerative medicine. For instance, the generation of organoids—miniature artificial organs that replicate the structure and functions of real ones—has become possible through the modification of stem cells. This opens the way to personalized disease models and the study of individual drug responses [doi.org/10.1016/j.cell.2016.05.082].
Genetically modified cells have become the foundation of many scientific advances of recent decades. They are used across a broad range of work—from basic research to the development of new treatment strategies and the production of pharmaceuticals. In the future, these developments will clearly play a key role in treating currently incurable diseases and in creating personalized medicine approaches.
The specialists at DUOX BIOTECHNOLOGIES have extensive experience in generating genetically modified cells. Contact us, and we will help you choose the best method of cell transgenesis for your tasks.
Applications of genetically modified cells
Disease modeling and investigation of molecular mechanisms
One of the most important applications of genetically modified cells is the creation of models of human diseases. For example, cells can be modified to mimic pathologies associated with specific genetic mutations, such as cystic fibrosis, Alzheimer’s disease, or cancer [doi.org/10.1016/j.tibtech.2011.04.009]. This makes it possible to study these diseases in vitro and to develop new treatment approaches.
Gene therapy
Genetically modified cells are also used in disease therapy. The most prominent example is CAR-T cancer therapy. This method involves the genetic modification of a patient’s T lymphocytes so that they express chimeric antigen receptors (CARs) capable of recognizing and destroying cancer cells. This approach has shown excellent results in the treatment of certain forms of leukemia and lymphoma [doi.org/10.1126/science.aar6711].
Biopharmaceuticals
Genetically modified cells are widely used in the production of biologic drugs. For example, engineered CHO cells are extensively used to produce antibodies and other protein therapeutics that are applied in the treatment of diseases such as cancer and rheumatoid arthritis.
Regenerative medicine and organoid generation
Another promising area is the use of genetically modified cells in regenerative medicine. For instance, the generation of organoids—miniature artificial organs that replicate the structure and functions of real ones—has become possible through the modification of stem cells. This opens the way to personalized disease models and the study of individual drug responses [doi.org/10.1016/j.cell.2016.05.082].
Genetically modified cells have become the foundation of many scientific advances of recent decades. They are used across a broad range of work—from basic research to the development of new treatment strategies and the production of pharmaceuticals. In the future, these developments will clearly play a key role in treating currently incurable diseases and in creating personalized medicine approaches.
The specialists at DUOX BIOTECHNOLOGIES have extensive experience in generating genetically modified cells. Contact us, and we will help you choose the best method of cell transgenesis for your tasks.