In mice receiving HPE-GNPs, the clinical score was significantly reduced compared to control, free-GNP and HPE (control 4.56±0.11 vs. HPE-GNP1: 2.94±0.26, HPE-GNP2: 2.44±0.38; p<0.01, HPE-GNP3: 1.83±0.41; p<0.001, HPE: 2.9±0.43; p<0.05) (Figure 2A). Here, GNPDA1 is linked to holoprosencephaly.