@theuncutpodcast_: As its sickle cell awareness month this month we want to remind you of the importance of donating blood. One donation can save up to 3 adult lives and 6 children’s lives ❤️ EP. 294 #sicklecell #nhsblooddonation #donatingbloodtosavelife
this is so good to know! definitely will be looking into this org, my dad recently found out he has sickle cell trait so we've been learning about it recently!
2025-09-09 13:58:53
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La Maison DeLaFontaine :
There are essentially two main curative approaches for sickle cell disease (SCD):
Bone marrow (or stem cell) transplantation
This is currently the only widely established cure.
The patient’s defective bone marrow (which produces sickled red blood cells) is replaced with healthy stem cells from a compatible donor (often a matched sibling).
These donor stem cells then produce normal red blood cells.
Limitation: Finding a well-matched donor is difficult, and the procedure carries risks (like graft-versus-host disease, rejection, or infections).
Gene therapy / genome editing
Instead of replacing the marrow, the patient’s own stem cells are collected, genetically modified in a lab, and then put back.
Two strategies are being used:
Gene addition: inserting a healthy copy of the hemoglobin gene or boosting fetal hemoglobin production (which prevents sickling).
Gene editing (like CRISPR-Cas9): directly correcting or modifying the defective hemoglobin gene (HBB) or turning on other protective genes.
The FDA and EMA have already approved the first CRISPR-based treatment for SCD (Casgevy) in 2023/2024.
2025-09-09 17:25:01
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Fashionwig :
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2025-09-09 16:12:33
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