Evaluating the Terrain: Prospects and Challenges of Cell and Gene Therapies
Gene therapy represents a pioneering medical strategy aimed at manipulating an individual's genetic material to prevent or treat diseases. It involves the introduction, modification, or substitution of genes within the patient's cells. The primary goal is to correct genetic abnormalities, replace missing genes, or regulate gene activity, thus addressing the root causes of various health conditions. This is achieved by delivering specific genetic material, typically DNA, into the patient's cells using various techniques such as viral vectors or gene-editing tools like CRISPR-Cas9. Gene therapy holds promise in addressing a wide range of medical conditions, including genetic disorders like cystic fibrosis and muscular dystrophy, as well as acquired diseases and certain types of cancer. One of the significant advantages of gene therapy is its ability to provide sustained gene expression following a single treatment, eliminating the need for continuous administration of drugs