How does gene therapy work?

How does gene therapy work?

• A. By introducing foreign genes into the genome
• B. By correcting defective genes responsible for disease development
• C. By generating mutated genes for research
• D. By cloning healthy cells from the individual

Answer: (B)

Gene therapy primarily functions by correcting defective genes that are responsible for the development of diseases. This approach aims to address the underlying genetic defects that contribute to the illness rather than merely alleviating symptoms. For instance, if a specific genetic mutation is identified as the cause of a disease, gene therapy can introduce a normal version of the gene, repair the defective gene, or use techniques like CRISPR to edit the gene at the molecular level to restore its proper function. This method has the potential to provide a more permanent and effective solution to genetic disorders, as it targets the root cause rather than just managing the symptoms. In contrast to other options, this approach emphasizes the restoration of normal gene function to prevent or cure disease. While introduction of foreign genes can happen in gene therapy, this is usually part of the broader context of correcting a defective gene rather than an end goal itself. Generating mutated genes for research has a different objective, aimed at understanding disease mechanisms rather than treatment. Cloning healthy cells, although useful in certain experimental contexts, does not directly address the genetic defects causing diseases as gene therapy does. Therefore, focusing on the correction of defective genes captures the essence of how gene therapy operates.

Gene therapy primarily functions by correcting defective genes that are responsible for the development of diseases. This approach aims to address the underlying genetic defects that contribute to the illness rather than merely alleviating symptoms. For instance, if a specific genetic mutation is identified as the cause of a disease, gene therapy can introduce a normal version of the gene, repair the defective gene, or use techniques like CRISPR to edit the gene at the molecular level to restore its proper function.

This method has the potential to provide a more permanent and effective solution to genetic disorders, as it targets the root cause rather than just managing the symptoms. In contrast to other options, this approach emphasizes the restoration of normal gene function to prevent or cure disease.

While introduction of foreign genes can happen in gene therapy, this is usually part of the broader context of correcting a defective gene rather than an end goal itself. Generating mutated genes for research has a different objective, aimed at understanding disease mechanisms rather than treatment. Cloning healthy cells, although useful in certain experimental contexts, does not directly address the genetic defects causing diseases as gene therapy does. Therefore, focusing on the correction of defective genes captures the essence of how gene therapy operates.