Gene Therapy

Gene therapy is the introduction of a gene into a patient’s cells to treat or prevent a disease caused by a mutated gene.

The introduced gene may be a functional copy of the mutated gene, or it may be a different gene that helps counter the effects of the mutation.

The therapeutic gene can be delivered directly into the patient’s body through in vivo therapy.

Alternatively, ex vivo therapy involves removing cells from the patient, genetically modifying them in the laboratory, and then transplanting them back into the patient.

The therapeutic gene is delivered using a vector. This vector is most commonly a genetically modified virus that enters the target cell and delivers the therapeutic gene for expression.

Some vectors integrate the therapeutic gene into the cell’s genome, allowing long-term expression of the introduced gene.

Other vectors keep the therapeutic gene outside the chromosomes as episomal DNA, which can remain active without integrating into the genome.

The vectors also contain promoter regions that enable transcription of the introduced gene into messenger RNA.