Gene Therapy Cure for 18 ‘Bubble Baby’ Patients
Nov 18, 2014 16:34:18 GMT -6
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Post by blackcatmagic on Nov 18, 2014 16:34:18 GMT -6
Researchers at UCLA announced today that they had cured 18 children who were born with the so-called Bubble Baby disease, a genetic disorder that leaves the young sufferers without a working immune system, putting them at risk of death from infections, even the common cold.
A team led by Dr. Donald Kohn, a stem cell researcher at the university’s Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research in Los Angeles, developed the breakthrough that cured 18 children who had adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID).
“All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems,” Kohn, a professor of pediatrics and of microbiology, immunology and molecular genetics in the life sciences at UCLA, said in a statement.
Kohn spent more than 30 years of research on finding the cure, UCLA said today when it announced the cure. Kohn and his team tested two therapy regimens on the children over the course of two multi-year clinical trials since 2009, UCLA said.
During the trials, the children's blood stem cells were removed from their bone marrow and genetically modified to correct the defect. All of the 18 patients -- who ranged in age from 3 months to 4 years at the time of treatment -- were cured without any side effects, UCLA said.
A team led by Dr. Donald Kohn, a stem cell researcher at the university’s Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research in Los Angeles, developed the breakthrough that cured 18 children who had adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID).
“All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems,” Kohn, a professor of pediatrics and of microbiology, immunology and molecular genetics in the life sciences at UCLA, said in a statement.
Kohn spent more than 30 years of research on finding the cure, UCLA said today when it announced the cure. Kohn and his team tested two therapy regimens on the children over the course of two multi-year clinical trials since 2009, UCLA said.
During the trials, the children's blood stem cells were removed from their bone marrow and genetically modified to correct the defect. All of the 18 patients -- who ranged in age from 3 months to 4 years at the time of treatment -- were cured without any side effects, UCLA said.
This is AMAZING!
