In humans, this can lead to genetic blood disorders such as thalassaemia, and possibly cancer. (newscientist.com)
«This step opens up an opportunity to take cells from patients with genetic blood disorders, use gene editing to correct their genetic defect, and make functional blood cells,» said Ryohichi Sugimura, a doctor at Boston Children's Hospital and lead author of one of the studies. (seeker.com)
Recent advances «give us reasonable hope that gene therapy will enter the armamentarium» to treat genetic blood disorders, some cancers, and inherited diseases, Fischer said, adding: «We might be very close to approval by regulatory authorities.» (sciencemag.org)