Gene Therapy

Gene therapy is a technique used to treat or prevent diseases by the insertion, replacement reparation, regulation or removal of genes within an individual’s cells and biological tissues.

The most common approach to gene therapy is insertion, by which a functional gene is inserted into the genome to replace a disease-causing mutated gene. Another method is modifying the gene sequence by substituting a mutated gene through homologous recombination.

Selective reverse mutation is a type of gene therapy in which an abnormal gene is repaired and returned to its normal function. Gene regulation involves the “turning on or turning off” of a particular gene to alter its functionality.

Types of gene therapy include germ line gene therapy and somatic gene therapy. In germ-line gene therapy, germ cells (i.e. sperms or eggs) are modified by the insertion of functional genes. These genetic changes would be heritable and theoretically passed on to future generations. In somatic gene therapy, genes are transferred into somatic cells, effecting only the patient with no risk to passing on the altered genes to offspring.