One particular type of SCID involves mutations in the ADA gene, and over the last couple of decades scientists have been investigating ways to deliver normal functioning versions of this gene into ...

The condition is informally known as the "bubble-boy" disease, and it involves mutations in the ADA gene resulting in a dysfunctional immune system. The experimental gene therapy involved ...

We investigated the long-term outcome of gene therapy for severe combined immunodeficiency (SCID) due to the lack of adenosine deaminase (ADA), a fatal disorder of purine metabolism and ...

On this episode, Gene talks to Ada about how her identity is always in flux, why she thinks poetry matters right now with the world on fire, and how both grief and freedom are animating this ...

If gene therapy works in ADA-deficient children, it may work in other diseases. The master cells of the immune system, T cells are sentries that alert the rest of the army to start making ...

High concentrations of unmethylated protein (>200 molecules per cell) inhibit ada gene activation by the methylated form, and will consequently switch off the adaptive response in the absence of ...

Mutations in the ADA gene mean the body doesn’t make enough of the enzyme to successfully do its job. This deficiency of ADA leads to a condition called severe combined immunodeficiency (SCID). Those ...

A treatment now pending approval in Europe will be the first commercial gene therapy to provide an outright cure for a deadly disease. The treatment is a landmark for gene-replacement technology ...

is caused by mutations in the ADA gene that creates the enzyme adenosine deaminase, which is essential to a functioning immune system. For children with the condition, even day-to-day activities ...

The drug was never approved in the U.S. ADA-SCID is a condition marked by a mutation in the gene making the adenosine deaminase (ADA) enzyme, which is essential for maintaining normal white blood ...

The treatment is an ex vivo gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), and is the second gene therapy to ever be approved in Europe after ...