Therapies for blood genetic diseases can be divided into different categories, including chemotherapy, radiotherapy, gene therapy, and hematopoietic stem cell transplantation. Among these treatments, gene targeting is progressively becoming a therapeutic alternative that offers the possibility of a permanent cure for certain blood genetic diseases. In recent years, gene therapy has played a more important role in curing genetic blood disorders. RNA interference (RNAi) is one of the directions for gene therapy, which was intensively studied in the past decades for its potentials in the treatment of diseases. In order to provide useful references and prospective directions for further studies concerning RNAi-based gene therapy for blood genetic diseases, current RNAi-based gene therapies for several typical blood genetic diseases have been summarized and discussed in this chapter.
Part of the book: RNA Interference