Gene targeting

Definition

Gene targeting is a technique used to alter specific genes within an organism's genome, allowing researchers to create mutations or insertions in a precise manner. This method is particularly valuable for studying gene function and modeling diseases, as it enables the investigation of genetic contributions to various biological processes. By manipulating genes in animal models, scientists can gain insights into gene functions that can ultimately lead to advancements in gene therapy and biotechnology applications.

5 Must Know Facts For Your Next Test

1. Gene targeting techniques often use homologous recombination to introduce specific changes into the genome, such as knocking out or modifying genes.
2. This approach is widely used in creating transgenic animals, particularly mice, which are crucial for studying human diseases and developing new treatments.
3. The success of gene targeting depends on the efficiency of delivering the targeting vector into the target cells, typically via methods like electroporation or viral vectors.
4. In gene therapy, targeted modifications can be designed to correct genetic disorders by repairing defective genes or replacing them with functional copies.
5. Ethical considerations around gene targeting include potential unintended consequences and the implications of creating genetically modified organisms, which continue to spark debate in science and society.

Review Questions

• How does gene targeting contribute to our understanding of gene function in animal models?
  • Gene targeting allows researchers to specifically manipulate genes within animal models, enabling them to observe the direct effects of these alterations on biological processes. By creating knockout mice or other genetically modified organisms, scientists can investigate how the absence or alteration of a particular gene influences phenotypes and contributes to diseases. This understanding is crucial for unraveling the complexities of genetic functions and identifying potential therapeutic targets for various health conditions.
• Discuss the role of CRISPR-Cas9 technology in advancing gene targeting techniques and its implications for research.
  • CRISPR-Cas9 has revolutionized gene targeting by providing a more efficient and accessible method for precise genome editing compared to traditional approaches. This technology allows researchers to easily create targeted mutations or modifications with higher accuracy and fewer off-target effects. The implications are significant, as CRISPR-Cas9 can accelerate research in genetics, facilitate the development of new therapies for genetic disorders, and enable breakthroughs in agricultural biotechnology by enhancing crop traits.
• Evaluate the ethical considerations surrounding gene targeting and its potential impact on future biotechnological advancements.
  • The ethical considerations surrounding gene targeting are complex and multifaceted, including concerns about unintended consequences, biodiversity, and the moral implications of modifying living organisms. As gene targeting becomes more prevalent in research and applications such as gene therapy and agriculture, discussions around responsible use, regulatory frameworks, and public acceptance become increasingly important. Evaluating these factors is essential for ensuring that biotechnological advancements benefit society while minimizing risks associated with genetic modifications.

Related terms

Knockout Mouse: A genetically engineered mouse in which a specific gene has been completely inactivated or 'knocked out', allowing researchers to study the effects of losing that gene.

CRISPR-Cas9: A revolutionary genome-editing technology that enables precise modifications of DNA sequences, making gene targeting more efficient and accessible.

Homologous Recombination: A process by which genetic material is exchanged between similar or identical DNA molecules, often used in gene targeting to replace or modify specific genes.