Genome-edited stem cells dodge antibodies, raising hope for chemo-free transplants

Stem cell transplantation (also called bone marrow transplantation) and gene therapy are among the most powerful curative approaches for blood diseases such as sickle cell disease, b-thalassemia, immune deficiencies and some blood cancers.

Replacing or correcting the blood-forming stem cells can offer the possibility of long-lasting benefit or a cure.

However, before patients can receive these therapies, they usually need intensive and often toxic chemotherapy or radiation to clear space in the bone marrow for the new stem cells. ...