How does gene therapy work?

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Our genetic code carries a set of instructions for producing proteins in the body. Any change in this code due to mutation results in a significant change in the production, structure or function of proteins- this affects certain physiological processes within the body. The underlying principle in gene therapy is that -the genetic code of a person can be altered to recover the functions of critical proteins.

Gene therapy can rectify genetic alterations in the following ways:

  • Gene transfer therapy– In this method, new genetic material is introduced into the cells. For example, if an altered gene inhibits protein formation, gene transfer therapy can introduce either a duplicate of the normal gene or a different gene that can produce proteins to help the cell function normally.
  • Genome editing– This new technique works by introducing gene-editing tools that can alter the existing DNA in the cell. Through this technique, one can add or remove the genetic material and also, alter the genome at precise locations. The best example of genome editing tool is CRISPR-Cas9.

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