A gene therapy for a rare type of severe combined immunodeficiency (SCID) was safe and sufficiently restored T-cell immunity in an early trial, results from a phase I/II study showed. At a single ...

David Vetter, the famous “bubble boy” of the 1970s, had to live in a plastic pod, eating sterilized food and wearing sterilized clothes, to avoid the everyday germs that can be fatal to those with ...

Please provide your email address to receive an email when new articles are posted on . A majority of boys with X-linked severe combined immunodeficiency experienced T-cell recovery and infection ...

NEW ORLEANS -- A next generation genetic therapy appeared to restore the immune system of patients with "bubble boy disease," but without leading to leukemia as a side effect, researchers reported ...

After a decade of unsuccessful attempts to correct one form of severe combined immunodeficiency using gene therapy, success has finally been achieved in another genetic type. Whether this success is ...

“I remember asking the doctor if my daughter was going to die,” said Chelsea Oakley, mother of Cora Oakley, a newborn who was diagnosed with ADA-SCID when she was just seven days old, in April 2017.

Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune cells, provide nonspecific immunity for infants early in life. The work stems ...

For decades, scientists have been trying to find ways to cure disorders that can be traced back to an error in one gene. One of the first diseases that researchers ...

Gene therapy is superior to haploidentical hematopoietic stem cell transplantation (HSCT) to treat X-linked severe combined immunodeficiency (SCID-X1), according to a report published online April 13 ...

UCLA researchers are restarting a study to give a gene therapy to kids with an ultra-rare disease. While the one-time therapy is safe and effective, the drug industry has been uninterested. The ...

Later this spring, a little girl in California who essentially has no immune system will receive a lifesaving treatment for “bubble boy disease” thanks to the persistence of a dogged group of parents, ...

A second-generation gene therapy for X-linked severe combined immunodeficiency (SCID-X1) repeats the efficacy demonstrated in a previous clinical trial, but without the adverse event of leukemia that ...