�y��vlog

A rare, aggressive form in children results from a mutation in a gene for a metabolic enzyme called adenosine deaminase 2.

The disease at issue is severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID.

As of last year, according to Scientific American, the FDA had approved nine gene therapies, which for the most part target rare physiological disorders, such as adenosine deaminase deficiency and lipoprotein lipase deficiency.

In 2016, a drug called Strimvelis was approved in Europe for the treatment of a condition called adenosine deaminase severe combined immunodeficiency, or ADA-SCID, a form of the disease that afflicted David Vetter.

We evolved a tRNA adenosine deaminase to operate on DNA when fused to a catalytically impaired CRISPR-Cas9.