Could gene therapy be used to replace neurons lost to Huntington’s disease?

Written by Alice Bough (Future Science Group)

Researchers from Jinan University (Guangzhou, China) and Pennsylvania State University (PA, USA) have collaborated on a study utilizing gene therapy to reprogram striatal astrocytes into GABAergic neurons. Their approach improved motor function in a mouse model of Huntington’s disease (HD). The authors of the paper, published in Nature Communications, suggest that this astrocyte-to-neuron conversion approach could be utilized as a new therapy for treating HD and other neurodegenerative disorders. “We are developing a series of NeuroD1-based gene therapies to reprogram brain internal glial cells directly into functional new neurons to treat a variety of brain disorders including HD, Alzheimer’s disease,...

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