Geneticists are working toward “gene therapy,” the replacement of defective genes with normal genes, to harness the selective properties of DNA and the tools available to essentially cure genetic diseases.
The ultimate goal of geneticists is to replace defective genes with normal ones. This treatment of genetic diseases at the most basic, intracellular level is known as “gene therapy. Gene therapy is one of the ideal treatments that scientists have long dreamed of, offering the possibility of curing genetically determined diseases once and for all. However, it is a very complex and precise process to realize. The first and most important step in ‘gene therapy’ is to identify the abnormal gene. To do this, scientists use the unique properties of DNA.
DNA is a molecule that consists of a double helix structure with two strands twisted into a helix. This structure was first identified by James Watson and Francis Crick in 1953 and is considered to be the fundamental structure that contains the genetic information of living things. The double helix is made up of four bases (A, T, C, and G), which are arranged in pairs in a specific way. If an A base is present on one strand, it will always be paired with a T base on the other side, and a G base will always be paired with a C base. This selective nature of base pairing allows scientists to identify genes that cause inherited diseases.
The first tool used to find genes is a small portion of a single strand of DNA. This piece of DNA, called a “probe,” consists of a couple dozen bases whose location on the chromosome is known. Probes play an important role in the experimental process. Thanks to their single-stranded nature, they can bind to the part of the DNA on the opposite strand that corresponds to their sequence of bases. The joining of two strands of DNA in this process is called hybridization. Hybridization is a crucial process in genetics, used to determine the exact location of genes, which can then be used to identify the causes of various genetic diseases.
A second important tool is a method called gel electrophoresis. This method utilizes the fact that the molecules that make up living things, such as proteins and nucleic acids, are charged. In an electric field, each molecule shows a unique migration pattern, which allows the molecules to be separated. Gel electrophoresis is an excellent method for separating and analyzing DNA fragments in particular. This process is an essential step in DNA research and gene therapy studies, allowing for a clear analysis of the structural properties and variations in DNA.
Armed with these tools, geneticists set out to track down the genes that cause inherited diseases. The study of inherited diseases is an area of great interest in modern medicine, with a key goal of pinpointing the causes of inherited diseases and developing ways to treat them. Geneticists first used gel electrophoresis to analyze the suspected genes responsible for inherited diseases. In this process, they used probes taken from healthy individuals to identify differences between normal people and those with the disease. These probes were made radioactive or fluorescent so that they would react with DNA fragments taken from patients in crossbreeding experiments. Because the part of the gene involved in the inherited disease is sequenced differently from that of a normal person, it will not hybridize with the probe. The results of these experiments have allowed geneticists to identify links between specific genes and inherited diseases.
Although this process is complex and time-consuming, it paves the way for us to cure genetic diseases at the source. Curing genetic diseases is no longer the stuff of science fiction. Gene therapy has recently emerged as one of the most prominent examples of cutting-edge medical technology, and these advances have given us new hope that we can cure diseases that were previously considered hopeless. In particular, gene therapy is offering new ways to treat incurable diseases such as cancer, genetic defects, and even degenerative diseases. Scientists are now going even further, exploring the possibility of preventing or even curing diseases through innovative methods such as gene editing technology.
In the coming years, research related to gene therapy will continue to advance, and it will make a significant contribution to solving many of the disease problems facing humanity. These advances in research and technology are not just limited to treating genetic diseases, but are closely linked to advances in healthcare in general. For example, advances in personalized medicine are based on genetic analysis technologies and will enable more effective and safer treatments by tailoring treatments to an individual’s genetic characteristics. Advances in genetics are playing a vital role in protecting human health and life, and there is much to look forward to in the future.
