CRISPR-Cas9 is a genome editing tool that enables geneticists and medical researchers to edit parts of the genome by removing, adding, or altering sections of DNA. It is a family of DNA sequences found in the genomes of prokaryotic organisms such as bacteria and archaea, which are derived from DNA fragments of bacteriophages that had previously infected the prokaryote. The Cas9 enzyme uses CRISPR sequences as a guide to recognize and open up specific strands of DNA that are complementary to the CRISPR sequence. The Cas9 enzyme and a guide RNA together form the basis of the CRISPR-Cas9 technology that can be used to edit genes within organisms.
CRISPR-Cas9 has a lot of potential as a tool for treating a range of medical conditions that have a genetic component, including cancer, hepatitis B, or even high cholesterol. However, much research is still focusing on its use in animal models or isolated human cells, with the aim to eventually use the technology to routinely treat diseases in humans. There is also a lot of work focusing on eliminating "off-target" effects, where the CRISPR-Cas9 system cuts at a different gene than the one that was intended to be edited.
Some key points about CRISPR-Cas9 include:
- It is faster, cheaper, more accurate, and more efficient than other genome editing methods.
- It is a unique technology that enables geneticists and medical researchers to edit parts of the genome by removing, adding, or altering sections of DNA.
- The Cas9 enzyme uses CRISPR sequences as a guide to recognize and open up specific strands of DNA that are complementary to the CRISPR sequence.
- CRISPR-Cas9 has a lot of potential as a tool for treating a range of medical conditions that have a genetic component.
- Much research is still focusing on its use in animal models or isolated human cells, with the aim to eventually use the technology to routinely treat diseases in humans.
- There is a lot of work focusing on eliminating "off-target" effects, where the CRISPR-Cas9 system cuts at a different gene than the one that was intended to be edited.
