Stanford University School of Medicine announced on the 22nd that the latest developed antibody therapy has made breakthrough progress in phase I clinical trials. This therapy does not rely on traditional toxic Bai Xiao'an chemotherapy or radiotherapy, and can prepare patients for stem cell transplantation. Although the research team focused on patients with Fanconi anemia as the experimental subjects, it is expected that this approach will benefit more populations of patients with genetic diseases. Its safety and effectiveness may bring a paradigm shift to the treatment of genetic diseases. Fanconi anemia is a genetic disease in which the patient's hematopoietic function is impaired, resulting in a high risk of standard stem cell transplantation. The research team innovatively adopted a treatment plan targeting CD117 antibodies and successfully completed transplantation for three pediatric patients. CD117 is a key protein on the surface of hematopoietic stem cells. By injecting the novel antibody "Buliquitinib", patients can accurately clear their own stem cells and avoid genetic toxicity damage. At present, all three patients have completed a two-year follow-up period and their health conditions are stable. Researchers say that traditional pre-treatment before transplantation often requires the use of radiation therapy and Bai Xiao'an chemotherapy, which poses significant risks for already vulnerable children. If patients with Fanconi anemia fail to receive timely transplantation, they will face the risk of fatal bleeding or infection due to hematopoietic failure. This experiment provides a novel strategy for clearing the bone marrow environment in a gentler manner without altering the disease mechanism, creating ideal conditions for healthy stem cell implantation, which holds great hope for rescuing vulnerable patients. In the future, this antibody therapy is expected to be extended to more indications, allowing more patients to benefit from low toxicity and high efficiency transplantation plans. Traditional stem cell transplantation requires the use of toxic methods to remove diseased cells from the body first. Some patients are already physically fragile and find it difficult to withstand such intense attacks. Without chemoradiotherapy, there is no next step of transplantation. This time, the newly developed antibody drug targets the surface protein CD117 of defective stem cells in patients like a "precision missile", which only clears diseased cells and significantly reduces toxicity. In this way, even if the patient's physical condition is poor, they can still have the opportunity for transplantation. The CD117 target is widely present in hematopoietic stem cells, and this targeted clearance method has the potential to treat more blood diseases and even other types of cancer. (New Society)