CRISPR Gene-Editing Therapy

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Introduction to Gene-Editing Therapy: CRISPR

CRISPR is a relatively new medical advancement that was inspired by the natural defense mechanism found in some microbes, such as bacteria. To protect themselves against invaders like viruses, these microbes capture snippets of the intruder’s DNA and store them away as segments called CRISPRs, or Clustered Regularly Interspersed Short Palindromic Repeats. If the same germ tries to attack again, those DNA segments (turned into short pieces of RNA) help an enzyme called Cas find and slice up the invader’s DNA.

In the laboratory, the CRISPR process consists of two main components: a DNA-cutting enzyme called “Cas9” modified with an added guide RNA. The guide RNA is engineered to mirror the target DNA of the gene to be edited. The guide RNA seeks for and locates, the target DNA. When the guide RNA finds an exact match, the Cas9 enzyme cuts, or modifies the target DNA.

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