| Cystic fibrosis (41) |  |
| Down syndrome (35) | |
| Genetic disease (68) | |
| Muscular dystrophy (42) | |
| Thalassemia (5) | |
| Cystic fibrosis (41) | |
| Down syndrome (35) | |
| Genetic disease (68) | |
| Muscular dystrophy (42) | |
| Thalassemia (5) | |
They wanted to edit the gene responsible for B-thalassemia, a fatal blood disorder. CRISPR (pronounced like`crisper')has swept through the scientific world in the last few years
They attempted to delete a gene for a blood disorder called beta thalassemia from the DNA of 86 nonviable embryos.
and after Chinese researchers reported they had employed indeed successfully the CRISPR/Cas9 system on nonviable human embryos to remove a part of the gene that causes a genetic blood condition called beta thalassemia.
and thalassemia who require treatment with regular transfusions and for whom it is difficult to find compatible donors.
Two patients with beta-thalassemia, a genetic disorder which normally requires regular blood transfusions, have been able to forgo transfusions for at least five months following a gene therapy treatment from bluebird.
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