Non-viral Gene Editing of Marrow Stem Cells Shows Potential in Early Study

Non-viral Gene Editing of Marrow Stem Cells Shows Potential in Early Study

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Non-viral Gene Editing of Marrow Stem Cells Shows Potential in Early Study

A non-viral, CRISPR-based gene-editing tool showed a potential to reverse mutations that cause sickle cell disease (SCD) by successfully editing genes in stem cells within the bone marrow of mice. Data from this proof-of-concept study also found that changes in stem cell genes were sustained for more than one year after a single course of treatment. “This new data supports the possibility of delivering a safer solution to treat blood disorders, including sickle cell disease,…

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