Gene Therapy: Biology, Ethics, and Medical Applications

Introduction

Gene therapy is the process of transplanting normal healthy genes into cells that are defective or missing in order to correct or cure genetic disorders. Gene therapy has been a controversial topic for many years. It was developed as a way to fight disease. This paper will focus on the biological process, the social and ethical implications, and a personal perspective on gene therapy.

Biological Basis of Gene Therapy

Gene therapy is also known as genetic engineering of dysfunctional genes. Genetic medicine is the broader field of medicine that it falls under. This area covers screening, diagnosis, prevention, and treatment of hereditary conditions in people. Gene therapy may offer a solution to problems found during genetic screening and has expanded beyond its initial discovery.

Life begins with a single cell that multiplies into trillions of cells, each having a specific function. These structures are made up of DNA, also known as the blueprint of each human being's makeup. DNA is deoxyribonucleic acid that carries hereditary information that guides development and functioning.

Defective or missing genes within cells have the potential to cause a multitude of different diseases. The intention of gene therapy is to introduce corrected genes into the body by replacing these missing or corrupt genes. This has shown effectiveness in treating and, in some cases, curing diseases. The genes introduced may be genetically engineered or naturally occurring.

The structure of genes has been manipulated since the 1970s through a process called splicing. Splicing involves cutting out part of the DNA in a gene and adding in new DNA. The scientist then inserts the gene into the new DNA, and the result is called genetically engineered recombinant DNA.

Types and Methods of Gene Therapy

There are two basic types of gene therapy: germ-line therapy and somatic cell gene therapy. Germ-line therapy is a preventative process where genes are introduced into reproductive cells or embryos in an attempt to correct any defects in future generations. The more commonly used somatic cell therapy is the process of inserting healthy genes into tissue or cells in an effort to produce naturally occurring correction. This type of therapy is also used in the form of viruses, as viruses attack their host and introduce their genetic material into the host cell. The targeted cells are the only ones affected, and the changes are specific to that individual.

Gene therapy is now being tested for treatment of major diseases such as AIDS, sickle-cell anemia, many types of cancer, and inherited diseases. Although scientists know the location and sequence of a chromosome, the exact outcome of manipulating genes is not yet fully predictable.

Gene therapy has proven to be a great discovery in many treatments. However, it does not come without complications, though these complications are rare. The hazard and unpredictable use of viruses are an area of concern. There is no specific way to ensure that the virus is inserted into the exact gene of interest, and there are chances that the virus will integrate or spread to another gene. Several studies have been conducted, and research continues to lower the possibility of complications.

Social and Ethical Implications

Gene therapy has been a complex subject since its discovery in the 1970s. The first approved human clinical study was not initiated until the 1990s. There are many social and ethical concerns regarding gene therapy. It is difficult to decide which disorders and traits should be studied.

Gene therapy was invented to treat and cure diseases. However, scientific studies have also discovered that gene therapy may treat "undesirable traits." These may consist of physical, mental, or psychological traits, such as eye color, learning disabilities, or personality. Using gene therapy for the enhancement of traits could benefit the individual but could also impact society. This could cause society to change the way it views what is now considered normal traits.

The advantages of gene therapy are that it can treat, cure, or prevent a known hereditary illness in an individual. If scientists decide to use it for future generations, gene therapy can also prevent future generations from inheriting certain diseases or traits. The disadvantages are that when viruses are used, there is no way to predict or control the actual outcome. Another major concern about gene therapy is that if we began using it to change physical, mental, or psychological traits, it will be the beginning of people wanting to enhance or control traits that should be left alone.

Both society and ethical frameworks maintain that gene therapy should not be used in any way other than for illnesses or diseases that cause suffering, disabilities, or possible death. Society has debates both for and against gene therapy, but when used for medical purposes, it has more support.

Conclusion

Gene therapy is an astonishing discovery and has the potential to lower the prevalence of many diseases we face daily. It has the possibility to treat, prevent, and possibly cure many terminal illnesses. Research continues constantly to find new and improved ways to deliver genes. Gene therapy is still considered experimental since there is no predictable outcome. The intention is to eliminate genetic disorders at the source of defect. For centuries, the challenge has been to find ways to use gene therapy that are fair, workable, and ethical for its use.