Gene therapy

Definition

Gene therapy is a medical technique that modifies genes to treat or prevent disease. It often involves inserting, altering, or removing genes within an individual's cells using vectors like viruses.

5 Must Know Facts For Your Next Test

1. Gene therapy can utilize viral vectors such as adenoviruses and retroviruses to deliver genetic material into cells.
2. There are two main types of gene therapy: somatic gene therapy, which targets non-reproductive cells, and germline gene therapy, which targets reproductive cells.
3. CRISPR-Cas9 is a groundbreaking tool used for precise editing of DNA in gene therapy applications.
4. The FDA has approved several gene therapies for conditions like spinal muscular atrophy and certain types of inherited blindness.
5. Potential risks of gene therapy include immune reactions, insertional mutagenesis, and ethical concerns surrounding germline modifications.

Review Questions

• What are the primary differences between somatic and germline gene therapy?
• How do viral vectors work in the context of gene therapy?
• What role does CRISPR-Cas9 play in modern gene therapy?

Related terms

Viral Vector: A tool commonly used to deliver genetic material into cells during gene therapy.

CRISPR-Cas9:

A precise genome-editing technology that allows for specific modifications in DNA sequences.

Insertional Mutagenesis:

An unintended alteration in a genome due to the insertion of new genetic material, potentially causing mutations.