A decade after gene therapy, children born with deadly immune disorder remain healthy

Good news! And this is only the beginning of realizing the enormous potential of gene therapy!

When will finally the sneeze gene be permanently removed? Probably, too late for me!

"Over a decade ago, UCLA physician-scientists began using a pioneering gene therapy they developed to treat children born with a rare and deadly immune system disorder. They now report that the effects of the therapy appear to be long-lasting, with 90% of patients who received the treatment eight to 11 years ago still disease-free.

ADA-SCID, or adenosine deaminase–deficient severe combined immunodeficiency, is caused by mutations in the gene that creates the ADA enzyme, which is essential to a functioning immune system. For babies with the disease, exposure to everyday germs can be fatal, and if untreated, most will die within the first two years of life. ..."

From the paper's abstract:

"... These patients were monitored in a long-term follow-up protocol over 8 to 11 years. Nine of 10 patients have sufficient immune reconstitution to protect against serious infections and have not needed to resume ERT or proceed to secondary allogeneic HSCT. ERT was restarted 6 months after GT in the oldest patient who had no evidence of benefit from GT. Four of 9 evaluable patients with the highest gene marking and B-cell numbers remain off immunoglobulin replacement therapy and responded to vaccines. ..."

A decade after gene therapy, children born with deadly immune disorder remain healthy | UCLA UCLA researchers provide update on patients treated for ADA-SCID between 2009 and 2012

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency