If the normal gene replaces the mutant allele, there is a chance that the transformed cells will proliferate and produce enough normal gene product for the entire body to be restored to the undiseased phenotype. For example, cancer cells contain faulty cells which are different from the normal cells and have defective proteins. Gene therapy is the treatment of disease by replacing, altering, or supplementing a gene that is absent or abnormal and whose absence or abnormality is responsible for the disease. Although the viruses that are used as vectors are disabled so that they cannot replicate, patients may suffer an immune response. the efficient transfer and expression of a variety of human genes into target cells, has already been accomplished in several systems. Gene therapy is the process of replacing defective genes with healthy ones, adding new genes to help the body fight or treat disease, or deactivating problem genes. Another concern associated with gene therapy is that it represents a form of eugenics, which aims to improve future generations through the selection of desired traits. Although some successes have been reported with gene therapy trials in humans—for example, with patients who have severe combined immunodeficiency (SCID) or hemophilia—significant technical challenges…. Your email address will not be published. For more information about gene therapy, login to BYJU’S. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear geno… Scientists have also explored the possibility of combining gene therapy with stem cell therapy. The researchers treated 11 patients, two of whom later developed a leukemia-like illness. Issues. Improved gene therapy systems are being developed by using nanotechnology. Ever since the dawn of mankind, diseases have plagued humans over the ages. In 1999 American teenager Jesse Gelsinger died after having taken part in a gene therapy trial. - Gene therapy today is effective against only a single gene disorder, and in multi-gene disorders like high blood pressure, Alzheimer’s, etc., gene therapy is yet to be developed. A promising application of that research involves packaging genes into nanoparticles that are targeted to cancer cells, thereby killing cancer cells specifically and leaving healthy cells unharmed. Gene therapy is a medical therapeutic process involving the delivery of the gene into cells to treat diseases. Essay on Human Gene Therapy 1696 Words | 7 Pages. The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. Germline gene therapy is not legal in many places as the risks outweigh the rewards. The Nemours Foundation - For Parents - Gene Therapy and Children. Be on the lookout for your Britannica newsletter to get trusted stories delivered right to your inbox. The first gene therapy was successfully accomplished in the year 1989. Some critics are particularly concerned about the safety of germline gene therapy, because any harm caused by such treatment could be passed to successive generations. Basically, there are two types of gene therapy. The first peer-reviewed journal in the field of human gene therapy, providing all-inclusive coverage of the research, methods, and clinical developments that are driving today's explosion of gene therapy advances. Kara Rogers is the senior editor of biomedical sciences at Encyclopædia Britannica, where she oversees a range of content from medicine and genetics to microorganisms. Essay on the Importance and uses of Gene Therapy for Human! Among human, gene therapy has been considered depending upon severity of diseases, which included deficiency like ADA and PNP, urea cycle defeats, or Lesch-Nyhan syndrome. When a normal gene is inserted into the nucleus of a mutant cell, the gene most likely will integrate into a chromosomal site different from the defective allele; although that may repair the mutation, a new mutation may result if the normal gene integrates into another functional gene. Learn about approaches to and issues surrounding gene therapy. Human gene therapy Gene therapy is a technique used in attempts to cure or prevent genetic diseases at the molecular level (basically at the source) by correcting what is wrong with defective genes, a good version of the defective gene … This has led to lower mortality rates and longer lifespans. It is an artificial method that introduces DNA into the cells of human body. The technique can be applied to target specific affected tissues in the body. The damaged proteins are replaced in the cell by the insertion of DNA into that cell. Your email address will not be published. Prerequisites for gene therapy include finding the best delivery system (often a virus, typically referred to as a viral vector) for the gene, demonstrating that the transferred gene can express itself in the host cell, and establishing that the procedure is safe. The first gene therapy was successfully accomplished in the year 1989. Basically, a normal allele is inserted into a patient's defective cells. Some aspects of gene therapy, including genetic manipulation and selection, research on embryonic tissue, and experimentation on human subjects, have aroused ethical controversy and safety concerns. Save 50% off a Britannica Premium subscription and gain access to exclusive content. To others, such a view of gene therapy legitimates the so-called medical model of disability (in which disability is seen as an individual problem to be fixed with medicine) and raises peoples’ hopes for new treatments that may never materialize. Premium Membership is now 50% off! The process involves introducing a healthy DNA into the cells responsible for producing reproductive cells, eggs or sperms. On the other hand, others have argued that genetic engineering may be justified where it is consistent with the purposes of God as creator. Required fields are marked *. One strategy is to replace a defective gene with its normal counterpart, using methods of recombinant DNA technology. Gene therapy is the fairly new method of disease treatment aimed mostly at genetic diseases. Safe … Benefits, however, would also be passed on indefinitely. Please select which sections you would like to print: Corrections? 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