Germ-line gene therapy

Definition

Germ-line gene therapy involves modifying the genes in germ cells (sperm or eggs) to correct genetic defects that can be passed on to future generations. It aims to treat or prevent disease at the earliest possible stage, before an embryo develops.

5 Must Know Facts For Your Next Test

1. Germ-line gene therapy is inheritable, meaning changes made will be passed down to offspring.
2. It targets germ cells such as sperm, eggs, or early embryos.
3. This type of therapy is controversial and heavily regulated due to ethical concerns about altering human genetics permanently.
4. Techniques used include CRISPR-Cas9 for precise gene editing.
5. Currently, germ-line gene therapy is not widely practiced and remains mostly experimental.

Review Questions

Related terms

Somatic Gene Therapy: Involves altering genes in specific tissues of an individual without affecting their offspring.

CRISPR-Cas9:

A powerful tool for editing genomes, allowing researchers to alter DNA sequences and modify gene function.

Gene Editing: The process by which DNA is inserted, deleted, modified or replaced in the genome of a living organism.