Reference no: EM133485831

BAYLOR MADDOX: Gene therapy is a medical treatment regarding the defective genes in a person's body, it aims to alter the bad genes in order to treat or prevent disease/illnesses. It can be classified as a type of genetic engineering that helps to repair defective genes or replace them with new genes to fight off diseases. Gene therapy can be provided to the patient by using a modified or inactive virus to deliver the new gene to the patient's cells and replace the faulty gene, as seen in the video. Another way that gene therapy can be administered is by using a gene editing tool that can specifically alter the patient's DNA. Electroporation is another method that is successful in replacing the patient's genes. It uses an electric field that opens up pores in the cell membranes which allows for the new genes to enter in. I have heard of gene therapy, but I was not very familiar with it or how it works. Some pros of gene therapy include: It can treat or prevent diseases, which gives the patient permanent relief from symptoms associated with their disease, it can correct genetic mutations, and it also has the ability to be personalized to the patient and their needs. A few cons, however, of gene therapy include the cost ($2.8 million), unknown side effects or complications that could be associated, unknown long-term effects, or possibly toxicity to the patient which could lead to further complications. Gene therapy has been used in patients who have Cystic Fibrosis (CF). Cystic fibrosis is a genetic disorder that affects mainly the lungs. Patients with CF lack, or have malfunctioning cystic fibrosis transmembrane conductance regulator (CFTR) protein which causes a buildup of thick mucus in the lungs that makes it extremely difficult to breathe. The drug associated with gene therapy used in patients with CF is called Trikafta. Trikafta is a combination of 3 different drugs that work simultaneously to aid in regenerating the CFTR protein. This drug has been tested and has proven to be effective in improving lung function in patients with CF.