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• The article discusses the potential of gene therapy to treat genetic diseases, its current challenges and the progress that has been made in developing successful treatments.
• It explains how gene therapy works, including identifying the target gene, inserting a healthy copy of it into cells, and introducing it into patients.
• Lastly, it highlights the importance of further research to better understand and improve existing treatments.


Gene therapy is an experimental medical treatment that uses genes to treat or prevent diseases caused by genetic mutations. It involves replacing a defective gene with a healthy one in order to correct the disease-causing mutation. This type of therapy has shown promising results for treating conditions such as cystic fibrosis, haemophilia A and B and some forms of cancer.

How does Gene Therapy Work?

Gene therapy works by targeting a specific gene that is causing or contributing to a disease. A healthy copy of this gene is inserted into cells and then introduced into the patient’s body through various methods such as viruses or liposomes. Once inside the cell, this new gene can produce proteins which help reverse or stop the disease process that was caused by the mutated version of this gene.


Despite the promising results seen so far with certain types of genetic diseases, there are still significant challenges associated with using this type of treatment. These include finding suitable delivery methods for introducing new genes into cells and avoiding immune system rejection when introducing foreign genes into patients‘ bodies. Additionally, researchers must also identify potential side effects from these treatments before they can be used safely on humans.


Despite these challenges, researchers have made significant progress in developing successful treatments using this technique over recent years. There have been numerous clinical trials demonstrating positive results in treating certain types of genetic diseases including cystic fibrosis, haemophilia A and B and some forms of cancer; however these results still need to be replicated in larger trials before they become standard therapies for these conditions .


Gene therapy holds great potential for treating genetic diseases but much work needs to be done before it becomes widely available as a therapeutic option for patients suffering from these conditions . Further research is needed to better understand how these treatments work at a molecular level as well as improving existing delivery methods so that they are safe and effective for use on humans .