Gene (or genome) editing refers to various technologies that are used to alter genetic material. This technology is used as a new means to treat diseases including genetic disorders. Fanconi anemia is an inherited disease that affects bone marrow leading to decreased production of all blood cell types. In general, editing of CD34+ (hematopoietic) stem cells have proven more difficult than for fibroblasts or embryonic stem cells. Some advances have been achieved in this regard; however, the efficiency of gene editing for CD34+ stem cells remains less than that possible with lentiviral vectors.
The successes and failures that led to the state of stem cell therapy have provided a framework for other types of molecular therapies now being studied and advanced. An example of this begins with the development of a new type of therapy for the immunometabolic disorder, ADA-SCID. The therapy, strimvelis, was developed by scientists in Milan, Italy at the San Raffaele Hospital and Telethon Institute (TIGET). They accomplished this by combining the science of gene therapy with stem cell transplantation.