Answer to Question #53037 in Biology for Brian Park

Question

What are the five criteria that a disease needs to meet in order to be a good candidate for gene therapy?

Answer 1

1. A disease must be caused by the mutations in a certain gene that has been determined already.
2. The disease must not be caused by global mutations of genome (in example, chromosomal rearrangements).
3. The disease must be tissue specific.
4. Disease must not be connected with the development because irreversible disorders cannot be changed.
5. Normal (wild type) gene can be easily cloned and inserted into the vector.