Fig. 4: Treatment with Galns-encoding vectors counteracts dental pathology.
MPSIVA rats were treated intravascularly with 6.67 × 1013 vg/kg AAV9-Galns vectors at 4 weeks of age. a Incisors from 1-month-old WT and MPSIVA rats presented similar morphology. At two months of age, incisors of MPSIVA rats presented malocclusion, dental fragility, and enamel hypoplasia, while incisors of MPSIVA treated rats were similar to those of WT rats. b LIMP2 immunohistochemistry of incisor in mandible sections from 2-months-old WT (n = 2), untreated (n = 3), and AAV9-Galns-treated (n = 2) MPSIVA rats. Untreated MPSIVA rats presented a marked lysosomal distension and vacuolization in ameloblast and papillary cell layers. Treatment of MPSIVA rats with AAV9-Galns prevented ameloblast lysosomal pathology. Scale bars: 100 µm. EN enamel, AM ameloblasts, PL papillary layer, CT connective tissue. c Ultrastructural analysis of incisors of 6-months-old rats. Ameloblast cells from incisors of untreated MPSIVA rats presented large electrolucent vacuoles (arrows), which were absent in AAV9-Galns-treated MPSIVA ameloblasts. n = 2 animals/group. Scale bars, 5 µm. d GFP immunostaining in incisors 2 weeks after administration of AAV9-GFP vectors (6.67 × 1013 vg/kg) to 4-weeks-old rats. n = 3 animals/group. DP dental pulp, OD odontoblasts, DE dentine, MB mandibular bone. Scale bars, 200 µm. Source data are provided as a Source Data file.
