GENE therapy is actually a sophisticated extension of conventional medical therapy. Rather than treat a patient’s disease with drugs or surgery, the patient receives DNA. Several approaches may be used for correcting the defect:
- · Gene Insertion:
A normal healthy gene may be inserted into a non-specific location within the genome to replace a non-functional gene. This approach is the most common.
- · Gene Modification:
An abnormal gene may be swapped for a normal gene using recombination.
- · Gene Surgery:
The abnormal gene may be repaired through reverse mutation or by changing the defect to return the gene to the normal function.
- · Gene Regulation:
The degree to which a gene is turned on or off may be altered. For example, certain genes may regulate the process of production of proteins. Targets to change regulation of these genes are a possibility.[47]
