This approach aims to introduce a working, or functional, gene into the body to research if it can produce a needed protein.

1. 1Creating a working gene.
2. 2Building a therapeutic vector.
3. 3Determining eligibility.
4. 4Delivering the working gene.
5. 5Monitoring safety and efficacy.

Following 18 years of further research, the first gene therapy trial launched in 1990. A four-year-old girl named Ashanthi DeSilva underwent a 12-day treatment for a rare genetic disease known as severe combined immunodeficiency.

Gene therapy can be used to modify cells inside or outside the body. When it’s done inside the body, a doctor will inject the vector carrying the gene directly into the part of the body that has defective cells.

The idea of germline gene therapy is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known.

Gene therapy achieves this by adding a correct copy of the gene into the genome of the cells in the target organ or tissue, while gene editing alters the genome at a specific location to correct or alter the genetic sequence.

Potential Harms to Human Health

• New Allergens in the Food Supply.
• Antibiotic Resistance.
• Production of New Toxins.
• Concentration of Toxic Metals.
• Enhancement of the Environment for Toxic Fungi.
• Unknown Harms.
• Gene Transfer to Wild or Weedy Relatives.
• Change in Herbicide Use Patterns.

One specific concern is the possibility for GMOs to negatively affect human health. This could result from differences in nutritional content, allergic response, or undesired side effects such as toxicity, organ damage, or gene transfer.