Somatic-cell gene therapy

Definition

Somatic-cell gene therapy involves the insertion, alteration, or removal of genes within the somatic cells of a patient to treat or prevent disease. It does not affect the patient's germline and therefore is not passed on to future generations.

5 Must Know Facts For Your Next Test

1. Somatic-cell gene therapy targets non-reproductive cells, meaning changes are not heritable.
2. It can be used to correct genetic disorders by introducing functional copies of defective genes.
3. Adeno-associated viruses (AAVs) and lentiviruses are commonly used vectors in somatic-cell gene therapy.
4. Approval for clinical use requires rigorous testing for safety and efficacy through clinical trials.
5. Efficacy can be limited by immune responses against the vector or the therapeutic gene product.

Review Questions

• What type of cells does somatic-cell gene therapy target?
• Why are adeno-associated viruses (AAVs) often used as vectors in this form of gene therapy?
• What is one major limitation of somatic-cell gene therapy?

Related terms

Germline Gene Therapy: Alters genes in germ cells, making changes heritable.

Vectors: Vehicles such as viruses used to deliver therapeutic genes into cells.

CRISPR-Cas9:

A genome editing tool that can precisely alter DNA sequences within living organisms.