Sickle Cell Anemia is a vicious genetic affliction that affects more than 8 million people every year. Genetic advancements made with the gene editing technology CRISPR has the potential to treat or, hopefully, entirely cure individuals afflicted with the disease as shown in this recent study. By using CRISPR to modify the stem cells within the bone marrow of patients and repair the genetic mutation causing Sickle Cell mutation within them, the hope is that those stem cells can then replicate and overtake a majority of the marrow supplying the patient with new red blood cells. There are current methods to transplant healthy stem cells from a donor into someone affected with Sickle Cell, but by using the patient’s own stem cells and CRISPR technology, the need for finding a donor and risking rejection is no longer necessary and can significantly improve success rates for such a procedure.
The use of CRISPR technology to repair genetic ailments is an exceedingly promising venture. By using this gene editing technology to repair a patient’s own genetic mutation, the instilled obsolescence of donor marrow/stem cells would be a massive breakthrough. Hopefully by continuing to explore the editing of stem cells, a multitude of other genetic ailments can be cured without the need for transplantation.
Links:
https://www.ucsf.edu/news/2024/11/428941/novel-gene-therapy-trial-sickle-cell-disease-launches
https://curesickle.org/crispr-scd
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