A blast of nostalgia from my academic past

One in a while, I see echoes of what sprouted and grew from where I did my academic research (not my research per se). A NYT article (not paywalled) discusses a successful gene therapy treatment for ADA-SCID. That is a genetic  disease that leaves babies without a functioning immune system. Most of those babies die within a year of birth. 

The lab I did my dissertation research in was at the NIH, in the institute we called Giblets, the National Heart, Lung & Blood Institute. That lab worked a couple of other NIH labs that treated any human genetic disease. The targeted disease was ADA-SCID. The first treatment procedure with human patients was done in 1990. 

I was in one of the key labs, but not directly involved in developing and administering the first human gene therapy protocol. I worked on peripheral research developing improved means to transfer genes into sick people. 

Cora Oakley, had ADA-SCID, 

but now she's cured

Here we are 35 years later and an apparently practical ADA-SCID gene therapy has finally arrived on the scene. 

Discussion about the first treatment protocol:

it was extremely complex, extremely expensive 

and not practical for widespread use

Progress is being made and gene therapy is becoming increasingly practical. Other targeted diseases include (1) sickle cell disease and β‑thalassemia, which is treated by stem‑cell gene addition and gene editing using CRISPR technology, and (2) hemophilia A and B, which is treated using virus (AAV)-based liver-directed gene therapy to increase blood clotting factor activity reduce or nearly eliminate spontaneous bleeding and the need for regular clotting factor infusions. 

There's still a long way to go, but gene therapy has gone a heck of a long way since 1990. 

By Germaine: Arose from primordial giblets