Heathcare Treasure

Rare disorders , also known as orphan diseases , affect a small percentage of the population but can have devastating effects on those affected. Traditional treatment options for rare disorders have often been limited, leaving patients with few therapeutic options. However, recent advancements in cell and gene therapies have brought new hope to individuals with rare diseases. These innovative therapies hold the potential to transform the lives of patients, providing them with a chance for recovery and improved quality of life. Commonly used vectors in cell and gene therapies In the field of gene therapy , adeno-associated virus (AAV) vectors have emerged as a popular choice for delivering therapeutic genes to target cells. AAV vectors are efficient, safe, and have demonstrated promising results in various clinical trials. Their ability to target specific tissues and sustain long-term gene expression has made them particularly suitable for treating rare disorders such as beta-thalassem

Gene therapy is a rapidly growing field in the medical industry and is showing great promise in treating complex diseases. By changing the DNA of cells, scientists aim to cure diseases at the genetic level. This approach has been successful in treating a range of conditions, including cancer, genetic disorders, and viral infections. One of the biggest advantages of gene therapy is its potential to cure diseases that have traditionally been considered untreatable. For example, it has been used to cure severe combined immunodeficiency (also known as "bubble boy" disease), a genetic disorder that leaves people without a functioning immune system. This has been a game changer for patients who previously had no hope of recovery. AAV Vectors in Gene Therapy Among the various viral vectors used to transport the desired genes, the adeno-associated viral (AAV) vector gene therapy is the most secure and efficient one. The vector can maintain long-term gene and protein expression after