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The Goal of Gene Editing and How It Works


What is the goal of gene editing?

The goal of gene editing is to prevent and treat disease by making small, precise cuts to remove a sequence of deoxyribonucleic acid (DNA) to turn off a gene that is making a harmful protein, turn on a gene to make more of a needed protein, or fix a mutated gene. 

Gene editing is primarily used in the lab to better understand disease in humans. Scientists study how removing individual genes affects animals that share a significant amount of genetic code with humans (like mice). Because there are still many ongoing questions about the safety of gene editing and room for error with the technology, it has only been used in rare cases where no other options are available. For example, it is being investigated in sickle cell disease.

Discover the science of gene editing

Gene editing works by changing the code of existing genes. This is typically done by programming an enzyme called a nuclease (which can split strands of DNA) to detect and break up a specific sequence in the DNA. This break triggers the DNA to repair, sometimes with the aid of a “blueprint” from donor DNA. 

Some gene editing treatments can modify cells in the body, while others involve extracting and then treating cells externally before returning them into the body.

Nuclease is a naturally occurring enzyme that can break bonds in DNA, essentially cutting it like scissors. In gene editing, nucleases are programmed by scientists to be able to detect exact sequences of DNA, which then trigger DNA repair. Sometimes information is included on how to rebuild the DNA to override the harmful code that led to the disease.