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

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,...