Gene editing
Gene editing is a group of technologies that give scientists the ability to change an organism's DNA. These technologies allow genetic material to be added, removed, or altered at particular locations in the genome. Several approaches to gene editing have been developed. A recent one is known as CRISPR-Cas9, which is short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9.
Overview
The CRISPR-Cas9 system has generated a lot of excitement in the scientific community because it is faster, cheaper, more accurate, and more efficient than other existing gene editing methods. Gene editing is of great interest in the prevention and treatment of human diseases. Currently, most research on gene editing is done to understand diseases using cells and animal models. Scientists are still working to determine whether this approach is safe and effective for use in people.
Techniques
CRISPR-Cas9
CRISPR-Cas9 was adapted from a naturally occurring genome editing system in bacteria. The bacteria capture snippets of DNA from invading viruses and use them to create DNA segments known as CRISPR arrays. The CRISPR arrays allow the bacteria to "remember" the viruses (or closely related ones). If the viruses attack again, the bacteria produce RNA segments from the CRISPR arrays to target the viruses' DNA. The bacteria then use Cas9 or a similar enzyme to cut the DNA apart, which disables the virus.
Zinc Finger Nucleases (ZFNs)
Zinc Finger Nucleases (ZFNs) are artificial proteins that can be designed to target and cut specific sequences of DNA. They are made up of two parts: a zinc finger protein, which can be designed to bind to a specific DNA sequence, and a nuclease, which can cut DNA.
Transcription Activator-Like Effector Nucleases (TALENs)
Transcription Activator-Like Effector Nucleases (TALENs) are similar to ZFNs but use a different protein to bind to the DNA. TALENs can also be designed to cut a specific DNA sequence.
Applications
Gene editing is used in research to understand diseases and may be used in treatment to cure them. For example, it is used in cancer research to understand the genes that contribute to cancer growth. It may also be used in gene therapy to treat genetic diseases such as Cystic Fibrosis or Sickle Cell Anemia.
Ethical Considerations
Gene editing also raises ethical questions, particularly when used in human embryos. The changes made can be inherited by future generations and might have unforeseen effects. This is why there is a call for a global moratorium on gene editing in human embryos.
See Also
References
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Contributors: Prab R. Tumpati, MD