Stanford: A team of researchers at the Stanford University School of Medicine
has used a gene-editing tool known as CRISPR to repair the gene that
causes sickle cell disease in human stem cells, which they say is a key
step toward developing a gene therapy for the disorder. The team went on to demonstrate that the mended cells could make a
functioning hemoglobin molecule, which carries oxygen in normal red
blood cells, and then successfully transplanted the stem cells into
mice. The researchers say the study represents a proof of concept for
the repair of blood-borne genetic diseases, such as sickle cell disease
and thalassemia.
