![]() ![]() This process typically takes place at a specialized treatment center. In apheresis, the part of the blood containing stem cells is separated from the rest of the blood, which is returned back to the body. In the next step the blood stem cells are collected from a vein through a process called apheresis, which typically occurs on or around fifth day of mobilization and takes several hours. Mobilization and apheresisĪs a first step, medications are given to help the blood stem cells leave the bone marrow and enter the blood stream – this is known as mobilization. Importantly, gene therapies are often referred to as “one-time treatments” – but while the therapy itself is administered only once, the treatment process from start to finish is complex and has several steps that can take several months. There are many different approaches to ex vivo gene therapy, but the administration process to deliver those different therapies is the same across many modalities. bluebird bio’s gene therapies are introduced ex vivo. In gene addition therapy, a functional gene is introduced into cells either ex vivo – where the cells are modified outside the body and then transplanted back in, or in vivo – where the cells are modified inside the body. Gene addition treats diseases at the genetic level by adding genetic material to a person’s cells to compensate for a missing or faulty gene. Additionally, using a patient’s own stem cells eliminates the risk of graft rejection, which can occur with traditional allogeneic transplants when a recipient’s immune system recognizes a donor’s cells as foreign, although risks commonly associated with mobilization and conditioning still apply. First, there is no need to identify a matched donor, which can be very challenging as only 30% of patients have a genetically matched sibling donor. Autologous HSCT, which is when a patient receives a transplant using their own stem cells has several benefits over traditional stem cell transplants. These cells are very important, as they have the potential to develop into other types of blood cells. This process works by using a patient’s own blood – or hematopoietic – stem cells. Bluebird bio’s three distinct investigational gene therapies utilize gene addition combined with an autologous hematopoietic stem cell transplant (HSCT).
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