[ad_1]
American scientists say that with the help of HIV, they have a gene therapy that cured eight children with severe combined immunodeficiency, known as "bubble boy" disease.
The results of the research were published by the New England Medical Journal and performed at a hospital in Tennessee.
These children, born with little or no immunity, now have functional immune systems.
Children with this disease who have not received treatment should live in completely sterile conditions and generally die as babies.
Within gene therapy, the baby's bone marrow is removed and the defect corrected in its DNA immediately after birth.
The "correct" gene (used to treat) is inserted into a modified version of the HIV cell, a virus that causes AIDS.
Scientists have announced that most babies leave the hospital for a month.
Evelina Mamkarc, one of the study's authors, said in a statement: "These patients are already children, respond to vaccines and have an immune system that creates all the immune cells they need to protect themselves from infection as they explore the world around them and live normal. " lives. "
"These are the first patients cured with SCID-X1 syndrome," she added, referring to the most common type of severe combined immunodeficiency.
The patients were treated at St. Jude Children's Hospital of Research in Memphis as well as Beniof Children's Hospital in San Francisco.
What is this syndrome?
David Vether's case is perhaps best known for all cases of severe combined immunodeficiency (SCID), a disease that should not have direct contact with the outside world of the plastic chamber.
Wind, nicknamed "Boy in a Balloon" (Bubble boy) He was born in 1971 with this syndrome and died 12 years after unsuccessful bone marrow transplantation.
The wind was placed in a plastic insulation chamber 20 seconds after he was born at Houston Children's Hospital, where he lived until he was six years old when he received a special plastic suit made by NASA, the US Space Agency.
His parents have already lost a child of this disease before his birth.
What are the treatment options?
Third, the best method to treat this syndrome is bone marrow transplantation from a related donor. However, the St. John's Hospital says that more than 80% of these patients do not have these donors and rely on the stem cells of other donors.
With this procedure, the chances of cure for immunodeficiency are lower and this carries a greater risk of serious side effects as a result of treatment.
Earlier innovations in gene therapy have produced alternatives to bone marrow transplantation, but in some cases they have also included chemotherapy and have affected many other diseases, such as blood diseases, sickle cell anemia and thalassemia, such as a metabolic syndrome.
[ad_2]
Source link