Illustration from research for gene therapy.
The technology behind gene therapy is so promising that it could revolutionize how we treat genetic diseases!
Gene therapy is a promising field that has the potential to revolutionize the way we treat genetic diseases. It involves the insertion of a healthy gene into a person’s cells to replace or correct a defective gene. Gene therapy can also involve the addition of new genes to the body or the suppression of genes that are causing harm.c
Gene therapy is founded on the understanding that genetic mutations can be responsible for causing various diseases. Some of these mutations are inherited from our parents, while others occur during our lifetime due to environmental factors such as radiation, chemicals, or viruses. These mutations can affect the function of proteins and enzymes that are essential for our health, leading to various disorders such as cystic fibrosis, hemophilia, muscular dystrophy, and cancer.
To treat genetic disorders using gene therapy, scientists use a vehicle called a “vector.” Vectors are genetically engineered to carry therapeutic genes into cells. They need to be efficient at delivering genetic material into cells and avoiding triggering an immune response. There are several types of vectors, but the most commonly used ones are viruses.
Viruses are ideal vectors because they have evolved to efficiently enter cells and insert their genetic material into the host’s DNA. Scientists can modify viruses by removing the genes that cause disease and replacing them with therapeutic genes. The virus is then used to infect the patient’s cells and deliver the therapeutic genes.
Gene therapy can be performed inside or outside the body. In some cases, the vector is directly injected into the patient’s bloodstream or a specific tissue to target the affected cells. In other cases, cells are removed from the patient and modified in the laboratory before being returned to the patient’s body.
There is no doubt that gene therapy is a groundbreaking and potentially life-changing medical advance. It has the ability to correct diseases at the genetic level, which is something that has never been possible before. Although there are still some challenges to overcome, such as perfecting the delivery method of the therapeutic genes, gene therapy holds great promise for the future.
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