FDA moves closer to sickle cell cure that uses gene editing

The Food and Drug Administration may be one step closer to what could be the first approval of a drug that uses the groundbreaking gene-editing tool CRISPR.

The drug, called exa-cel, treats sickle cell disease, an inherited blood disorder that affects an estimated 100,000 people in the U.S., most of whom are Black, according to the Centers for Disease Control and Prevention.

The illness causes the body’s red blood cells, usually disk-shaped, to take on a crescent or sickle shape. When that occurs, the cells can clump together, leading to clots and blockages in the blood vessels. That may result in a variety of complications, including excruciating pain, trouble breathing or stroke.

AnUnusualRelic,
@AnUnusualRelic@lemmy.world avatar

And then you get malaria.

CosmicSploogeDrizzle,
@CosmicSploogeDrizzle@lemmy.world avatar

Oddly enough those with the sickle cell trait (one abnormal gene and one regular gene) actually have protection against malaria. Those with sickle cell anemia (2 abnormal genes) unfortunately, do not.

AnUnusualRelic,
@AnUnusualRelic@lemmy.world avatar

Oh, it’s more complicated than I thought.

Melatonin,

Maybe a stupid question but is that a real pic and if so why do the blood cells have veins? Or are those cracks? Either way, what’s up with those?

FlyingSquid,
@FlyingSquid@lemmy.world avatar

I don’t have sickle cell (I’m white so that would be highly unlikely), but I am a chronic pain sufferer, so I have always had a lot of sympathy for other people in chronic pain conditions and, based on the little I know about sickle cell, they reach 10 on the pain scale even more often than I do, which probably results in a lot of suicides. If there’s any cure for anyone in pain, I really want them to have it, not just to make them feel better and improve their lives, but to save their lives.

I have seen little, if any, discussion on the role chronic pain can play in suicides, but it’s something a lot of us have to struggle with because we see no other way out of feeling pain all the time.

I really hope for my brothers and sisters who have to suffer from this that relief is coming soon.

Icarus512,

Heartfelt and earnest response that makes me feel so shitty there isn’t anything to help alleviate what ails you. Hopefully you’ll get your cure soon enough as well.

FlyingSquid,
@FlyingSquid@lemmy.world avatar

I appreciate that, but I honestly care more about them than me. What I have is pretty rare. What they have is far more common. Far more people are suffering. I have medication that helps mitigate the issue and allows me to function basically normally. There are people with sickle cell that can’t get out of bed.

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