Gene therapy replaces defective genes with normal ones using viral vectors. It has been successful in treating diseases such as inherited retinal disease, thalassemia, and some cancers. Germline genetic engineering could modify the human genome, but has not been done yet.
Gene therapy is a way of inserting genes into a patient’s cells and replacing pre-existing alleles, or gene variants, to perform certain therapeutic functions. Until now it has mainly been used to replace defective mutant genes, or alleles, with normal alleles, but in theory it could be used to modify the human genome arbitrarily. If gene therapy were applied to reproductive cells in the gonads (the germline), these genetic changes would be heritable. This process has never been done, but it has a name: germline genetic engineering.
Since the early 1980s, gene therapy has been used to make medicines. Let’s say a human needs a certain protein as a medicine. This therapy uses a viral vector, i.e. a virus modified to contain the DNA to be introduced. Large amounts of the virus are injected into the target area, or sometimes tissue is removed, infected with the virus, and then re-implanted. Viruses are modified in such a way that the vast majority are unable to self-replicate independently, providing little chance for pathogenic infection. The virus introduced the new DNA into the genomes of human cells, in the same way that normal viruses introduce their genetic material into human cells, hijacking the cellular machinery.
After the new DNA is integrated into the target cell, the cell begins producing specified proteins from the new genetic material, which can be life-saving in some cases. For example, patients with severe diabetes can be given the cellular machinery to produce insulin, obviating the need for regular injections. The benefits of therapy can last for weeks, months, or even years or a lifetime.
Gene therapy has been used successfully to treat inherited retinal disease, thalassemia, cystic fibrosis, severe combined immunodeficiency, and some cancers. Medical miracles not possible with any other approach have been demonstrated by gene therapy, such as reprogramming the body’s natural sentinels, T cells, to attack cancer cells. Gene therapy holds promise for treating diseases such as Huntington’s disease and sickle cell disease. As the therapy continues to mature, it could save millions of lives.
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