Mol Ther Methods Clin Dev
March 2025
Recombinant adeno-associated virus (rAAV) is a widely used viral vector for gene therapy. However, a limitation of AAV-mediated gene therapy is that patients are typically dosed only once. In this study, we investigated the possibility of delivering multiple rounds of AAV through intracerebral injections in the mouse brain, and discovered a dose-dependent modulation of the second administration by the first-round AAV injection in a brain-wide scale.
View Article and Find Full Text PDFIn vivo astrocyte-to-neuron (AtN) conversion induced by overexpression of neural transcriptional factors has great potential for neural regeneration and repair. Here, we demonstrate that a single neural transcriptional factor, Dlx2, converts mouse striatal astrocytes into neurons in a dose-dependent manner. Lineage-tracing studies in Aldh1l1-CreERT2 mice confirm that Dlx2 can convert striatal astrocytes into DARPP32 and Ctip2 medium spiny neurons (MSNs).
View Article and Find Full Text PDF