RNAi-based gene therapy (RNA interference) is quickly becoming a hot topic in the world of therapies for blood diseases. Blood diseases are those defined as disorders in the hematopoietic system or plasma compounds. Therapeutic approaches for these diseases are divided into several categories, including chemotherapy, radiotherapy, hematopoietic stem cell transplantation, and RNAi-based gene therapy. Of these, RNAi-based gene therapy is progressively becoming an alternative offering the possibility of a permanent cure for some blood diseases.
Gene therapy attempts to treat inherited diseases using normal copies of the defective genes to correct a cellular dysfunction or provide a new cellular function. RNAi not only suppresses transcription by transcriptional gene silencing but it also activates a homology-based mRNA degradation process by post-transcriptional gene silencing, both of which result in the decrease of the coding transcript level (mRNA).
RNAi-based gene therapy possesses several therapeutic advantages, including less immunogenicity. This is in large part due to using non-protein-coding "gene products" to trigger RNAi, making gene therapy potentially less likely to be hindered by the host immune system. Another potential advantage is sequence specificity, which, when compared to traditional small molecules and protein drugs, in combination with the universal treatment spectrum make it an ideal treatment for blood genetic diseases.
One example of the advantage of RNAi-based gene therapy is seen through research conducted with myeloid leukemia. Many therapies have been explored as a cure for this disease, of which chemotherapy is always considered a frontline treatment, mainly containing cytotoxic agents and therapeutic molecules. Although the leukemia cells initially respond well to chemotherapy, it tends to lose some effectiveness after about 6-12 months. In addition, side effects of traditional cytotoxic agents arise, which limit its function with the progression of the disease. RNAi-based gene therapy has been explored by researchers in suppressing the growth and proliferation of myeloid leukemia cells. The results showed a significant decrease in the level of target proteins by limiting the expression of certain genes. RNAi-based gene therapy, when compared to treatments such as chemotherapy, would have a significant effect on the cells with the potential for a notable decrease in side effects.
Although a very exciting possible form of therapy for these types of diseases, there is much research to be done before determining whether RNAi-based therapeutics would be an efficient tool. It seems though that the therapy, in combination with other therapies, could be a very effective new way to treat blood genetic diseases.
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