Potential of gene therapy.
The goal of gene therapy research is to determine whether a new gene can be used to replace or inactivate a mutated gene to treat or possibly prevent a genetic disease. The diagrams below represent a section of a gene's DNA sequence. Researchers are testing a few methods of potential gene therapies, including:
Providing a healthy copy of a mutated gene that causes disease
Introducing a new gene into the body
Suppressing, or “knocking out,” a mutated gene that is functioning improperly