Genetic Engineering in the Press by GEG

Researchers could eventually help develop viable treatments and ultimately a cure for motor neuron disease (MND). They have identified biochemical changes in a protein that is affected by MND. TDP-43 is a protein found in every cell of the body but is particularly important for the health of motor neurons and the brain cells that control voluntary muscle movement. Two research projects have been run, using CRISPR and looking at how TDP-43 proteins become dysfunctional in motor neurons. Researchers found diseased versions of TDP-43 can damage healthy versions of the protein, which may create a cycle of protein dysfunction and degeneration over time. They also discovered that biochemical pathways which control neuron death are triggered early, even before MND symptoms begin. To change the course of the disease, pharmaceutical drugs are needed to be able to prevent neuron death and this TDP-43 protein dysfunction. Researchers are now treating genetically modified mice with MND with different pharmaceutical drugs that specifically target the underlying causes of the disease, and correct the disease mechanism. Their aim is to stop the TDP-43 degenerative cycle and halt the progression of the disease.