CRISPR moves from the lab to human trials, targeting blindness, beta thalassemia and sickle cell anemia

manufactured t cells
Image: Penn Medicine

It’s only been seven years since scientists first learned how to precisely and reliably splice the human genome using a tool called CRISPR, making it possible to think about snipping out disease-causing mutations and actually cure, once and for all, genetic diseases ranging from sickle cell anemia to certain types of cancer and even blindness.

Editas Medicine and Allergan recently announced a more acceptable form of gene editing… . They are enrolling patients born with a congenital vision disease into what will be the first test in the U.S. of whether CRISPR can fix a mutation in the cells of a living human body. Other ongoing trials, including one from partners Vertex Pharmaceuticals and CRISPR Therapeutics that is treating blood diseases, rely on treating patients’ cells outside of their body and introducing them back to the body… .

Related article:  Viewpoint: With reasonable regulation, we can turn wild plants into food with gene editing

Scientists agree that CRISPR holds great promise in giving researchers unprecedented power to snip out abnormal stretches of DNA. … CRISPR works well enough in the lab, in a dish of human cells, but as with any technology, there are glitches. … Then there is the bigger question of what longer term, unanticipated effects man-made edits to the human genome might have.

Read full, original post: CRISPR Gene Editing Is Being Tested in Human Patients, and the Results Could Revolutionize Health Care

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