blog




  • Essay / Genetics of diseases other than cancer - 1496

    Gene therapy is not a new field and has been evolving for decades. Even with the best efforts of researchers around the world, gene therapy has seen only limited success, primarily for common diseases like cancer. Will gene therapy make it possible to overcome incurable diseases? Gene therapy is an experimental technique that treats or prevents diseases through the use of genes. Scientists have focused on diseases caused by single-gene abnormalities such as sickle cell anemia, muscular dystrophy, hemophilia, and cystic fibrosis. A gene cannot be easily inserted directly into your cells, so it is delivered using a carrier called a vector. There are unresolved issues with this technology, including its ephemeral nature, human immune response, viral vector issues, multigene disorders, tumor induction, and cost. Although it carries risks, gene therapy shows great promise in overcoming the rarest diseases, with clinical successes recorded since 2006, including Leber congenital amaurosis and adrenoleukodystrophy. hemophilia and Parkinson's disease. Cystic fibrosis (CF) is a fatal genetic disease that causes many health problems (Fig. 1), also the most common fatal autosomal recessive disease in the global Caucasian population. It is caused by a point mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Most people have two working copies (alleles) of the CFTR gene, although only one is needed to prevent cystic fibrosis. With modern medicine, the life expectancy of people with cystic fibrosis has increased, but there is no definitive cure for the disease (fig.2). Gene therapy for cystic fibrosis has been tested in humans using liposomes and viruses. Currently, the observed effect lasts only a few...... middle of paper ......mined at a significantly lower rate than unmodified T cells, in a broader group of patients as well as plans to increase the persistence of more cells in the body to achieve a therapeutic effect. Gene therapy has had many failures and successes, but our knowledge of gene therapy and research in this area has led to significant advances in treatments and therapies. Gene therapy still has a very long way to go due to the dangers of the field, but it certainly has the potential to cure incurable diseases, thanks to its breakthroughs in the early stages of research. Gene therapy has already surpassed many other treatments in terms of effectiveness. As researchers delve deeper into this area of ​​potential, there is a good chance that incurable diseases will be cured, but it will still take many years of research and understanding...