An American charity has given an Oxford gene therapy company £500,000 to investigate possible treatments for motor-neuron disease.
Oxford BioMedica, based at Oxford Science Park, has been given the grant from the charity Andrew's Buddies/FightSMA, which is dedicated to the development of treatments for Spinal Muscular Atrophy (SMA), a form of motor-neuron disease that is one of the most common causes of death in childhood.
The disease, caused by a defect in a gene known as SMN-1, affects one in every 6,000 births. One in 40 people are carriers.
It leads to loss of muscle function. Children with the most severe form of the disease usually die before the age of two, when their lungs stop working.
Oxford BioMedica will use genetic engineering to replace the defective gene.
The company aims to produce a treatment which can be tested in hospital clinics within two years.
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