Sickle cell disease treated using CRISPR

[HAL9000]

NPR Cookie Consent and Choices

Key bit from the story...

For the experimental treatment, scientists remove cells from patients' bone marrow and use CRISPR to edit a gene, which enables the cells to produce a protein known as fetal hemoglobin. Fetal hemoglobin is made by fetuses in the womb to get oxygen from their mothers' blood but usually stops being produced shortly after birth.


The hope was that restoring production of fetal hemoglobin would compensate for the defective hemoglobin produced by sickle cell patients. Beta thalassemia patients don't have enough hemoglobin.


Scientists had hoped that after the treatment, which Gray received July 2, 2019, at least 20% of the hemoglobin in her system would be fetal hemoglobin.


Blood tests so far have shown the levels far exceeded that. About 46% of the hemoglobin in Gray's system continues to be fetal hemoglobin, the researchers reported. In addition, fetal hemoglobin has remained present in 99.7% of her red blood cells, they reported.


Another promising finding is that a biopsy of Gray's bone marrow cells found more than 81% of the cells contained the intended genetic change needed to produce fetal hemoglobin, indicating the edited cells were continuing to survive and function in her body for a sustained period.

Perhaps most importantly, the changes appear to have translated into significant health benefits for Gray. She hasn't had any severe pain attacks since the treatment and hasn't required any emergency room treatments, hospitalizations or blood transfusions.

NPR Cookie Consent and Choices

 

[kalidarkone]

Hmm its sounding hopeful, however this article seems to be more balanced and outlines potential risks.

Using CRISPR to cure sickle-cell disease at its root

Christopher Vakulskas, director of Enzyme Evolution at Integrated DNA Technologies explains how the company’s novel gene editing tools may one day provide single-treatment cures for diseases caused by single gene mutations, including sickle cell dise

www.epmmagazine.com