![]() What we have now found is that we can mimic TDP-43 function with a designer DNA drug, thereby restoring correct stathmin-2 RNA and protein level in the mammalian nervous system.” “Without stathmin-2, motor neurons disconnect from muscle, driving paralysis that is characteristic of ALS. Reduced TDP-43 activity causes misassembly of the RNA-encoding stathmin-2, a protein required for maintenance of the connection of motor neurons to muscle,” said Cleveland. ![]() “In almost all instances of ALS, there is aggregation of TDP-43, a protein that functions in maturation of the RNA intermediates that encode many proteins. In ALS, TDP-43 loss impacts the motor neurons that innervate and trigger the contraction of skeletal muscles, causing them to degenerate, eventually resulting in paralysis. The new study builds upon ongoing research by Cleveland and others regarding the role and loss of TDP-43, a protein associated with ALS, AD, and other neurodegenerative disorders. One such drug has been approved to treat a childhood neurodegenerative disease called spinal muscular atrophy. Several designer DNA drugs are currently in clinical trials for multiple diseases. Credit: Erik Jepsen, UC San DiegoĬleveland is broadly credited with developing the concept of designer DNA drugs, which act to either turn on or turn off genes associated with many degenerative diseases of the aging human nervous system, including ALS, AD, Huntington’s disease, and cancer. This was achieved using specially engineered DNA drugs that reinstate the normal processing of RNA that encodes proteins.ĭon Cleveland, Ph.D., Distinguished Professor of Medicine, Neurosciences, and Cellular and Molecular Medicine at UC San Diego School of Medicine, is among the most highly cited researchers in the world for his work investigating neurodegenerative diseases. In a study published in the journal Science, a group of researchers, led by Don Cleveland, Ph.D., who is a Distinguished Professor of Medicine, Neurosciences, and Cellular and Molecular Medicine at the University of California San Diego School of Medicine, reveal that the loss of stathmin-2 can be reversed. This displacement results in the loss of stathmin-2, a protein that is vital for the regeneration of neurons and the preservation of their links to muscle fibers, both of which are essential for muscle contraction and movement. In nearly all cases of amyotrophic lateral sclerosis (ALS) and in as many as half of all Alzheimer’s disease (AD) and frontotemporal dementia incidents, a protein known as TDP-43 is misplaced from its usual position in the cell’s nucleus. ![]() This breakthrough, specifically relevant to amyotrophic lateral sclerosis where this function is typically compromised, could potentially pave the way for clinical trials. In research involving both mice and humans, a genetically-engineered medication was found to replenish the levels of a critical protein, thereby preserving the function of motor neurons. In some diseases, such as ALS, those proteins are lost, the neurons die, and paralysis results. An illustration of the connections between muscle fibers and a motor neuron, the latter of which relies on crucial proteins to create and send signals to the fibers to contract, resulting in movement. ![]()
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