Gene therapy

Definition

Gene therapy is a medical technique that involves modifying or manipulating the genes within an individual's cells to treat or prevent disease. It often uses viral vectors to deliver therapeutic genes into target cells.

5 Must Know Facts For Your Next Test

1. Gene therapy can be used to correct defective genes responsible for disease development.
2. Viral vectors, such as adenoviruses and retroviruses, are commonly employed to deliver therapeutic genes.
3. Somatic gene therapy targets non-reproductive cells and does not affect future generations.
4. Germline gene therapy targets reproductive cells and can be passed on to offspring, though it is currently controversial and mostly prohibited.
5. CRISPR-Cas9 is a revolutionary tool used in gene editing that allows for precise modifications of DNA sequences.

Review Questions

• What role do viral vectors play in gene therapy?
• How does somatic gene therapy differ from germline gene therapy?
• What technology has significantly advanced the precision of gene editing in recent years?

Related terms

CRISPR-Cas9:

A genome editing tool that allows scientists to make precise changes to DNA.

Adenovirus: A type of virus commonly used as a vector in gene therapy due to its ability to efficiently deliver genetic material into cells.

Retrovirus: A class of viruses used as vectors in gene therapy that integrate their genetic material into the host cell's genome.