Crispr for genetic problems: SIckle cell disease

 

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the problem? It costs 2 million dollars for the treatment.

Much of this cost is because of the expenses of trails to get it approved.

Nature magazine reports that the companies are submitting the therapy to the FDA for approval.

Vertex and CRISPR Therapeutics have submitted their CRISPR-based ex vivo cell therapy exagamglogene autotemcel (exa-cel) for FDA approval, for sickle cell disease (SCD) and beta-thalassemia. A regulatory decision on the gene-editing candidate is expected within 8 to 12 months. The companies have also filed for approval in Europe and the UK.,

>more information here

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Forget about He Jiankui, the Chinese scientist who created gene-edited babies. Instead, when you think about gene editing you should think of Victoria Gray, the African-American woman who says she’s been cured of her sickle-cell disease symptoms.

Link to the Royal Society conference on this technique.

Youtube has the list to watch their lectures.