RNA Abnormalities in Human Diseases and How They Might Be Corrected

RNA molecules, and their regulation, are central to the functioning of human cells. Moreover, abnormalities in RNA regulation can lead to human disease. For example, in bone marrow failure diseases, such as dyskeratosis congenita, the telomerase RNA required for chromosome maintenance is abnormally degraded. Understanding this degradation mechanism has suggested possible therapies to reverse this process and provide treatment for this disease. Similarly, neurodegenerative diseases such as ALS and Alzheimer's disease involve alterations in proteins that regulate RNA, leading to RNA abnormalities in these diseases. Determining how RNA regulation is altered in these diseases is an important area of research, and determining how those alterations can be reversed may lead to new therapeutic approaches.