The FDA approved the world’s first therapy using gene-editing Crispr technology. Exa-cel — a treatment for sickle-cell disease developed by Vertex Pharmaceuticals and Crispr Therapeutics — works by genetically manipulating patients’ cells. Unlike medicines that treat diseases, Crispr targets their root causes, creating a potential one-and-done cure for diseases like cancer and diabetes. It’s poised to transform medicine, though its exorbitant cost (Exa-cel rings up at $2.2M/patient) is a roadblock. https://www.scientificamerican.com/article/fda-approves-first-crispr-gene-editing-treatment-for-sickle-cell-disease/#:~:text=On%20December%208%20the%20U.S.,blood%20cell%20shape%20and%20function. #Science is awesome 🧬🦾🚀🤯

Sam Mendoza

2 years ago

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The FDA approved the world’s first therapy using gene-editing Crispr technology. Exa-cel — a treatment for sickle-cell disease developed by Vertex Pharmaceuticals and Crispr Therapeutics — works by genetically manipulating patients’ cells. Unlike medicines that treat diseases, Crispr targets their root causes, creating a potential one-and-done cure for diseases like cancer and diabetes. It’s poised to transform medicine, though its exorbitant cost (Exa-cel rings up at $2.2M/patient) is a roadblock.

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#Science is awesome 🧬🦾🚀🤯

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FDA Approves First CRISPR Gene Editing Treatment for Sickle Cell Disease

Sickle cell disease causes red blood cells to take on a sickle-like shape, making them fragile and less able to transport oxygen. Most people with sickle cell disease who received a new gene editing