NeuroD1 + Dlx2 gene therapy

**NeuroD1 + Dlx2 gene therapy** is an investigational **AAV-based in vivo gene therapy** designed to regenerate striatal neurons by delivering the neural transcription factors **NeuroD1** and **Dlx2** to astrocytes in the brain, thereby converting endogenous striatal astrocytes into **GABAergic neurons**, including **DARPP32-positive medium spiny neurons**. The approach has been reported in preclinical Huntington's disease mouse models, where astrocyte-to-neuron conversion reduced striatal atrophy and improved motor phenotypes and survival. The technology is associated with **NeuExcell**, which has licensed related intellectual property from **Penn State** and has pursued development of astrocyte-to-neuron conversion programs for neurological disorders including Huntington's disease.

Other names

NeuroD1 + Dlx2 gene therapyNeuroD1 and Dlx2 gene therapyNeuroD-1 and Dlx2 gene therapyNeuroD 1 and Dlx2 gene therapyNeuroD1 + Dlx2 AAV gene therapy