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A research team led by the University of Oxford has found a promising treatment for degenerative disease spinal muscular atrophy (SMA) , a leading genetic cause of child death. Pip6a is highly effective at delivering SSOs to a wide variety of tissue in the body. Dr Suzan Hammond, Department of Physiology, Anatomy and Genetics - SMA occurs when people lack a gene called survival motor neuron 1 (SMN1). It can affect children in the womb or adults. This makes them unable to produce enough SMN protein, resulting is motor neurone degeneration and increasing muscle weakness. However, people have an almost identical gene called SMN2. Existing proposed treatments are based on altering SMN2 to include a crucial part that is found in SMN1, enabling the production of SMN protein.
