TAKING THE SICK OUT OF SICKLE CELL

This entry was posted by Sunday, 16 October, 2011
Read the rest of this entry »

MAKING SICKLE CELL GO AWAY

Mice. You can’t live with’em.  And you can’t get pissed off every time they get cured.

What is it THIS time? Well, it sounds pretty great, actually. Stuart Orking  of Harvard Medical School has been working with mice that have sickle cell disease which is a wretched, painful disease that can cause back pain, abdominal pain, skin ulcers, and a long list of other stuff including organ failure (yes, that’s fatal).  Dr. Orking’s mice (like humans with sickle cell)  make the wrong kind of hemoglobin -hemoglobin s – which causes red blood cells to become sickle shaped (sickle cell, get it?).

Isn’t this WHY we invented science? To DO something about bad things? Knowing just to know is nice. But doing good trumps just knowing over here at ScienceAintSoBad. I hope you understand.

So.

CURE

Oh yes! That’s what I said. Cure. Just for mice. But this is one that WILL lead to practical therapies. Here’s how it works.

Fetuses don’t get sickle cell. It’s only when they switch over to adult type hemoglobin (few months old)  that they – the babies with the bad gene –  start making  the wrong kind of hemoglobin. If they had stuck with fetal hemoglobin, they would have been fine. Fetal hemoglobin doesn’t lump up;  and it  moves oxygen around the body just like its grown up version does.

This is where Stuart Orking comes into the  the picture. Orking, also of Children’s Hospital Boston and Dana Farber (I wouldn’t want to leave anybody out), figured out how to get a protein called BCL11A to shut up. Once he silenced the protein, fetal hemoglobin reemerged and the mice were, basically, cured of their sickle cell disease. There’s no reason this shouldn’t work on humans. Now Orking has to figure out how to do this in a practical way – some type of drug, I imagine, – and do it on beings that have better health plans than mice.

IN THE MEANTIME

No need to despair. There are, currently, drugs that can help sickle cell disease. Not a cure. But, for many people, they do a lot of good. Take good care of yourself. Better days are coming.

—————-

Credit for the above image? Moi.


Leave a Reply