Prenatal stem cell therapy for inherited diseases: Past, present, and future treatment strategies

Abstract

Abstract Imagine the profits in quality of life that can be made by treating inherited diseases early in life, maybe even before birth! Immense cost savings can also be made by treating diseases promptly. Hence, prenatal stem cell therapy holds great promise for developing new and early-stage treatment strategies for several diseases. Successful prenatal stem cell therapy would represent a major step forward in the management of patients with hematological, metabolic, or immunological disorders. However, treatment before birth has several limitations, including ethical issues. In this review, we summarize the past, the present, and the future of prenatal stem cell therapy, which includes an overview of different stem cell types, preclinical studies, and clinical attempts treating various diseases. We also discuss the current challenges and future strategies for prenatal stem cell therapy and also new approaches, which may lead to advancement in the management of patients with severe incurable diseases. Significance statement This review summarizes the past, the present progress, and the future potential of prenatal stem cell therapy. Recent and previous studies are discussed, focusing on both preclinical and clinical data, highlighting both the drawbacks and the novel findings leading to the progress of prenatal stem cell therapies into the clinic.