Gene therapy involves modifying defective genes in order to cure a disease or help your body fight the disease better. Researchers have found that this can be done by replacing mutated genes. Certain cells become diseased because some genes do not work or work incorrectly. Replacing the defective gene can aid to treat certain diseases. Gene therapy can also involve fixing the mutated genes by turning them off so that they no longer promote disease and turning on healthy genes to prevent or inhibit disease. If your immune system doesn't attack diseased cells because it does not recognize them as trespassers, gene therapy can be used to train it to be more evident to threats.
Researchers are still studying how and when to use gene therapy. A recent study has found that a progressive treatment performed before birth may help children born with the rare genetic disorder, Pompe disease. Pompe disease is caused by mutations in a gene that make an enzyme that breaks glycogen down in cells. This causes glycogen to build up throughout the body. This condition is often treated right after birth with replacement enzymes. Although some babies are also born with an immune disorder that blocks the infused enzymes, for which the therapy is inhibited. This new treatment was injected into a mother's abdomen and then guided into the umbilical cord vein of the baby suffering from Pompe disease. After many biweekly infusions in utero, the baby was observed to be born healthy.
Even though this form of gene therapy had only been performed on one mother, I believe there is a lot of potential for doing more research on this topic. In so, it is not only the enzyme infusion that is the innovation, it is treating this problem earlier and while still in utero. This is because patients with such early-onset lysosomal storage diseases are ideal candidates for this type of prenatal therapy since organ damage starts in utero. If in-utero enzyme-replacement therapy continues to bring positive results, it will indeed save many lives.