The team used a modified version of HIV that can’t cause AIDS to deliver the correct gene into the DNA of their blood stem cells, replacing those that do not function correctly. SCID is the result of an immune system so highly compromised that it is considered almost absent. Jude Children's Research Hospital was trailed on eight infants who lacked a matched sibling donor. It is also known as the bubble boy disease and bubble baby disease because its victims are extremely vulnerable to infectious diseases and some of them, such as David Vetter, have become famous for living in a sterile environment. David Vetter inside his protective plastic bubble. Author: Michelle Goebel Instead, they tend to rely on blood stem cells from donors who are not family, a situation that is better than no treatment but often leads to marked side effects. Namun perjuangan David tidak sia-sia, kematiannya di usia 12 tahun berhasil membuka tabir baru mengenai penyakit langka ini. However, this means patients must have a matched sibling donor and less than 20 percent of patients usually do, according to the study authors. Texas, AS - Hampir seluruh hidup David dihabiskan di dalam sebuah gelembung ruangan steril karena ia memiliki penyakit tidak punya sistem kekebalan tubuh. The disease affects around 1 in 50,000 to 100,000 newborns, according to the NIH. The condition is caused by a mutation in the IL2RG gene, which is responsible for the body’s instruction manual for producing a protein essential for a healthy immune system.Ĭurrently, there is a viable treatment for SCID-X1: a bone marrow transplant. Without proper treatment and care, patients have little chance of living past infancy. The disorder is an inherited condition that almost always occurs in males. “This is a first for patients with SCID-X1.” Children born with the genetic condition known as ‘bubble baby disease’ have no functioning immune system, meaning that even a normally mild infection can be fatal. Jude Department of Bone Marrow Transplantation and Cellular Therapy in the US. The patients in the current trial “are toddlers now, who are responding to vaccinations and have immune systems to make all immune cells they need for protection from infections as they explore the world and live normal lives,” said study author Ewelina Mamcarz of the St.
0 Comments
Leave a Reply. |
AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |