Gene therapy
altering DNA within cells in an organism to treat or cure a disease
Gene therapy is a medical procedure that involves the introduction, removal, or alteration of genes within an individual’s cells and biological tissues. The main goal of gene therapy is to treat or cure genetic diseases by correcting or incorporating new genetic material into the individual’s DNA. This technology has the potential to revolutionize the way we approach treating genetic disorders and ultimately improving patients’ health outcomes.
There are several different approaches to gene therapy, including gene replacement, gene editing, and gene inhibition. Gene replacement therapy involves adding a healthy copy of a mutated or missing gene into the patient’s cells to compensate for the genetic defect. Gene editing therapy involves using specialized tools like CRISPR-Cas9 to repair or modify specific genes in the patient’s DNA. Finally, gene inhibition therapy involves targeting specific genes that contribute to the development of the disease and preventing their expression.
While gene therapy holds great promise, there are several challenges that must be addressed before it can be widely adopted. These include issues with targeting the specific cells that need to be treated, ensuring the safety of the therapy, and developing effective delivery methods. Nevertheless, with further research, gene therapy has the potential to provide safe and effective treatments for a wide range of genetic diseases, thereby improving the quality of life for millions of patients around the world.
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