For example, CRISPR is used to make “knockout” models of disease in a wide range of animals, enabling researchers to study the underlying genetic causes. Genome editing is widely used in studies in a variety of organisms. CRISPR/Cas9 works by cutting a DNA sequence at a specific genetic location and deleting or inserting DNA sequences, which can change a single base pair of DNA, large pieces of chromosomes, or regulation of gene expression levels. CRISPR was discovered through NIH-funded basic research on how bacteria defend themselves from viruses. Because CRISPR/Cas9 is an RNA-based system, it can be more efficiently and easily modified than the protein-based approaches and allows for targeting of multiple sites. An additional method is called clustered regularly interspaced short palindromic repeats, also known as CRISPR/Cas9.ĬRISPR/Cas9 is the most widely used genome editor and is a powerful tool for understanding gene function. Genome editing allows researchers to mimic this natural process of DNA repair.Īdvanced genome editing methods engineered from proteins include zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and meganucleases. Genome editing builds on an earlier discovery that a broken section of DNA in a gene triggers a cell’s repair mechanism to stitch together the break. While techniques to modify DNA have existed for several decades, new methods have made genome editing faster, cheaper and more efficient. Genome editing can be used to correct, introduce or delete almost any DNA sequence in many different types of cells and organisms.
Genome editing, also called gene editing, is an area of research seeking to modify genes of living organisms to improve our understanding of gene function and develop ways to use it to treat genetic or acquired diseases. Genome Editing in Research Genome Editing in the Clinic Ethical and Safety Concerns Genome Editing and NIH Funding Images Video Additional Resources
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