Sentences with phrase «bubble boy disease»

Often called «bubble boy disease,» SCID devastates the immune system and leaves patients vulnerable to lethal infections.

The team engineered their DNA scissors to target a mutation that causes severe combined immune deficiency (SCID), also known as «bubble boy disease,» an often fatal condition that disrupts the ability of immune T cells to fight infections.

He hopes that Cas9 - based therapies for T cell - related disorders, which include autoimmune diseases as well as immunodeficiencies such as «bubble boy disease,» will enter the clinic in the future.

The LCA2 trials are a rare success for the field of gene therapy, which has also cured children with the immune disorder known as bubble boy disease.

The first people to be treated with a gene therapy had ADA - SCID, also called «bubble boy disease», and some later got leukaemia, probably because the virus carrying the new genes also switched on cancer genes.

Not exact matches

The company is also seeking deeper inroads into rare diseases, with a number of assets in late - stage development, and last year it scored a major regulatory win when Europe approved its «bubble boy syndrome» gene therapy Strimvelis.

At six months Katlyn was diagnosed with «bubble boy» disease, formally known as severe combined immunodeficiency (SCID), which robs the immune system of the ability to fight infection.

2000 Announcement that two boys in France with X-linked SCID or «bubble boy» disease have been cured using gene therapy.

When scientists used gene therapy to cure «bubble boy» disease 7 years ago, the work was hailed as a milestone for the field.

Gene therapy's clearest success to date has been restoring the health of about 40 children with severe combined immunodeficiency (SCID), also known as «bubble boy» disease because patients can not fight off infections and are often isolated to protect them from germs.

A «miracle» gene therapy treatment for children suffering from the fatal «bubble boy» disease has been halted in France, after one of the patients developed leukaemia as a direct consequence of the treatment.

Old grudges bubbling up: that boy who said there wasn't room for her to join his club, the teacher that forgot her name, that lowdown group of girls who told everyone she had a bad disease.