What is gene therapy? How does it work?

What is gene therapy and how does it work?

• what is gene therapy and how does it work? which diseases or conditions could it be used to treat? Are there risks or side effects with the treatment? the condition is? who does it affect? what are the symptoms? what other treatments are available for it? where can you get more information? answer my questions please in simple sentences! thankyouu!

• Answer:

  Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene in order to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment. In gene therapy, DNA that encodes a therapeutic protein is packaged within a "vector", which is used to get the DNA inside cells within the body. Once inside, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patient's disease.