Gene-sequence swap using CRISPR to cure hemophilia

23 julio 2015

For the first time, chromosomal defects responsible for hemophilia have been corrected in patient-specific iPSCs using CRISPR-Cas9 nucleases. Hemophilia A occurs in about 1 in 5,000 male births and almost half of severe cases are caused by identified «chromosomal inversions.» In a chromosomal inversion, the order of the base pairs on the chromosome are reversed so the gene doesn’t express properly and the sufferer lacks the blood coagulation factor VIII (F8) gene, which causes blood to clot in healthy people.img src=»» height=»1″ width=»1″ alt=»»/