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