How can gene editing change the protein produced by a gene?

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Gene editing changes the protein produced by a gene by precisely altering the underlying DNA sequence, which serves as the blueprint for protein synthesis. Because the sequence of nucleotides in a gene determines the amino acid sequence of the resulting protein, modifying this code can directly impact the protein's structure and function.12

Researchers achieve these changes using tools like CRISPR-Cas9 or site-directed mutagenesis to introduce, remove, or replace specific segments of genetic material. These modifications can have several effects on the protein:123

  • Functional Alteration: By changing specific amino acids, scientists can modify how a protein functions, such as improving the properties of industrial enzymes or studying protein-receptor interactions.12
  • Loss of Function: Mutations can lead to the production of a functionless protein or the complete absence of a protein, which is often the target when correcting disease-causing genetic errors.142
  • Regulatory Changes: Editing the regulatory regions of a gene can alter when or how much of a protein is produced, rather than changing the protein's structure itself.4

These precise interventions allow scientists to dissect structure-function relationships and develop potential gene therapies to restore normal protein production in cases of genetic disease.12