What are the advantages and disadvantages of gene therapy for SCID?
severe combined immunodeficiency (SCID) treated with gene therapy has proved successful for the treatment of disease, but in some infants at the age of 90 caused leukemia. Four out of nine children treated with gene therapy for SCID in European experiments have developed blood cancer several years after treatment. Newer studies show that SCID gene therapy can be successful without causing cancer.
Gene therapy involves introducing a genetically changed virus called a vector into the bone marrow of the patient. The bone marrow sample is removed from a sick child before adding genes containing virus in the laboratory. After the changed bone marrow is re -introduced into the patient's body, it begins to create the missing genetic connection caused by the Scid.
The use of gene therapy for the disease ceased after four of the European children developed leukemia. One of the children died after leukemia treatment failed, causing controversy over gene therapy for SCID. Scientists found genetic material changedIn the laboratory, he disrupted the normal functioning of a nearby gene that causes cancer, but eight out of nine surviving patients recovered for live normal lives.
When the immune system does not work properly, the body cannot fight against viral or bacterial infection against common diseases. Without gene therapy for scid or bone marrow transplantation, most children die before their first birthday. Bone marrow transplantation represented the only affordable disorder treatment before scientists discovered gene therapy for SCID. Problems with bone marrow transplantation focused on finding suitable donors to reduce the likelihood of rejecting the body.
Before the existence of gene therapy for SCID, a child born with this disease was isolated to avoid exposure to the seeds. In the 1970s of the 20th century, the disorder received international attention when doctors reduced David Vetter to the sterile environment of birth pItem searching for a viable bone marrow donor. The child was referred to as a boy in a bubble, which caused a disease to be called Bubble Boy syndrome.
David Vetter died in 1984 after receiving a bone marrow transplant from his older sister. Its bone marrow partially corresponded to its siblings, but the mutation caused the development of the Epstein-Barr virus. Scientists began experimenting with gene therapy for Scid after the boy's death. They found that using its own bone marrow eliminated the chance to reject the present in the bone marrow transplant.
After European children treated with gene therapy developed leukemia, scientists began to look for ways to improve the vector virus without causing cancer. Since 2011, new methods of gene therapy for SCID have been approved for test experiments. Human studies attempts include participants in the monitoring of the study for 15 years to measure the efficacy of new therapeutic methods.
Ten forms of sciid exist, identified by which cellsMissing in newborns. He considered himself a rare illness, being handed over to the descendants of parents who carry defective genes, with more affected boys than girls. Children born with a condition usually face death when infected with bacteria that cause diseases common in childhood.