What are the requirements a disease must meet for gene therapy to be an effective option for treatment?

Question

Select all that apply.
• It should involve many genes.
• Target cells must be found throughout the patient's body.
• It must have a genetic cause.
• Target cells must be accessible.

Answer 1

For gene therapy to be an effective option for treatment, the following requirements should be met:

• It must have a genetic cause. (Gene therapy specifically addresses diseases caused by genetic mutations or abnormalities.)
• Target cells must be accessible. (For gene therapy to work, the cells that need to be treated must be reachable, whether through direct injection, systemic delivery, or other methods.)

The other two options, "It should involve many genes" and "Target cells must be found throughout the patient's body," are not necessary for gene therapy to be effective. Gene therapy can target single-gene disorders, and it does not require target cells to be widespread throughout the body.