Base editing corrects mutation and slows motor neuron disease in mouse model

In a study published in Molecular Therapy Advances a team of researchers led by Professor Haruhisa Inoue (Center for iPS Cell Research and Application, Kyoto University) and Professor Yuishin Izumi (Tokushima University) demonstrate that adenine base editing can correct a disease-causing genetic mutation and alleviate neurodegeneration in hereditary motor and sensory neuropathy with proximal dominant involvement (HMSN-P), providing proof of concept for a precision gene therapy for inherited motor neuron diseases.

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