Around 1 in 50 people are affected by a genetic disorder in the U.S. This is a significant proportion of the population. Genetic disorders can cause severe health conditions incompatible with life, meaning people die from these disorders in the most severe cases. Because of this, scientists and researchers are doing everything possible to find a cure for these conditions. One cure that looks promising is gene therapy. If you want to find out whether gene therapy can be used to cure genetic disorders, keep reading below:
What Are Genetic Disorders?
There are currently over 6000 known genetic disorders in the world. A genetic disorder is a health issue that is caused by mutations in the genome. The mutation can be a chromosomal abnormality, or it can affect one gene or multiple genes or be a chromosomal abnormality. This type of disorder can be hereditary, meaning it is inherited from parents who carry a faulty gene, or it can occur spontaneously in the womb.
Some of the common types of genetic disorders include:
- Downs syndrome
- Cystic fibrosis
- Huntington’s disease
- Sickle cell anemia
- Duchenne muscular dystrophy
- Hemophilia
What is Gene Therapy?
In simple terms, gene therapy is a method that alters a person’s genes to cure or treat disease. This type of therapy can work in several ways, including:
- Deactivating a disease-causing gene that is not working correctly.
- Treating or curing disease by introducing a new or altered gene into the body.
- Swapping a disease-causing gene with a healthy replica of the gene
There are several different types of gene therapy. Here are some of the most promising ones:
- Viral vectors – scientists have discovered that viruses have a natural ability to transport genetic material to cells. In this case, a virus is modified to remove any infectious diseases before being used to carry therapeutic genes into human cells.
- Human gene editing technology – this type of gene therapy aims to repair mutated genes or disrupt harmful genes. One example of this is the PiggyBac transposon system.
- Patient-derived cellular gene therapy products – during this type of therapy, cells are obtained from a patient before being genetically altered and returned to the patient.
Can Genes Be Used to Cure Genetic Disorders?
At present, gene therapy cannot cure genetic disorders. However, the future of gene therapy looks promising. It is hoped that genetic therapies will be used in the next few years to treat, cure and prevent certain inherited disorders. As well as this, many scientists believe that they will also be used to treat and cure certain types of cancer or infections such as HIV.
A genetic disorder can occur for two reasons; because you have the incorrect amount of genetic material or because a mutation changes your genes. There are many genetic disorders; however, Downs Syndrome and Cystic Fibrosis are the most well-known. In the past, these disorders were untreatable, meaning people had to live with any side effects for life, but things are now starting to look up. In time, gene therapy is hoped to cure disorders such as Downs Syndrome.
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