What is gene therapy?

Gene therapy is a therapeutic approach that aims to address and treat the underlying cause of genetic diseases that currently have no cure. This approach allows healthcare providers to replace a mutated, disease-causing gene with a healthy copy, avoiding surgery or medications. By introducing a healthy copy of a gene into the system, gene therapy may restore the function of the protein and potentially eliminate the subsequent adverse effects that the mutated gene inflicts at the source.

Several approaches to gene therapy are currently being tested. Some of these approaches include:

  • Replacing a disease-causing mutated gene with a healthy copy of the gene.
  • Deactivating a misfunctioning gene.
  • Introducing a new gene into the body to modify the effects of the mutated gene.

Source: https://ghr.nlm.nih.gov/primer/therapy/genetherapy

Outside of gene therapy, other alternative approaches of manipulating genes to fight disease are emerging, such as gene splicing and antisense oligonucleotide work, which create even greater opportunity for management of these rare diseases.