en Cellular and molecular Neuroscience Cellular and molecular Neuroscience Original Original <div style="text-align: justify;"><strong>Introduction</strong>: Targeting smoothened (SMO) has been a remarkable strategy for treating sonic hedgehog (Shh)-dependent cancers, especially medulloblastoma. GDC-0449, also known as vismodegib, is a potent SMO inhibitor with mild toxicity and high affinity. Thus, this study aimed to investigate the anti-medulloblastoma efficacy of vismodegib in the DAOY medulloblastoma cell line.&nbsp;<br> <strong>Methods</strong>: Human DAOY medulloblastoma cells were cultured in Dulbecco&rsquo;s Modified Eagle&rsquo;s Medium (DMEM). 50, 80, 100, and 150 &mu;M doses of vismodegib were used to treat cells. 3-(4,5-dimethylthiazol-2-yl)-2,5-diphenyltetrazolium bromide (MTT), scratch, and trypan blue assays, as well as real-time polymerase chain reaction (RT-PCR) and immunofluorescence studies, were performed 24 and 48 h post-treatment.&nbsp;<br> <strong>Results</strong>: The MTT and trypan blue assays showed a significant difference in viability between the control and treatment groups. The results of the scratch assay showed that in the control group, the cells were able to repair the lesion, while the scratch disintegrated at higher doses of vismodegib. The expression of SMO, Gli1, and MYCN genes, the main components of the SHH signalling pathway, was significantly reduced in the vismodegib-treated groups compared to the control group. Also, a notable increase in the activation of metastasis-promoting genes (Bax and p53) and a reduction in the activation of the metastasis-inhibiting gene (Bcl2) were observed.<br> <strong>Conclusion</strong>: The results of the current study confirm that vismodegib is a potent inhibitor of the Shh pathway and may be used in combination with new therapeutic methods to treat medulloblastoma.</div> <span style="font-size:11pt"><span style="line-height:200%"><span calibri="" style="font-family:"><span style="font-size:12.0pt"><span style="line-height:200%"><span new="" roman="" style="font-family:" times=""><span style="color:black"><b><i></i></b></span></span></span></span></span></span></span> Vismodegib, Drug therapy, Medulloblastoma, Neoplasm, Toxicity 609 618 http://bcn.iums.ac.ir/browse.php?a_code=A-10-6423-1&slc_lang=en&sid=1 Masume Behruzi masumebehrouzi@gmail.com 13700319475328460054302 0009-0004-3554-2755 No Department of Anatomical Sciences, School of Medicine, Iran University of Medical Sciences, Tehran, Iran. Mehrdad Ghorbanlou mehrdad.ghorbanlou@gmail.com 13700319475328460054303 13700319475328460054303 No Department of Anatomical Sciences, School of Medicine, Iran University of Medical Sciences, Tehran, Iran. Ronak Shabani shabanironak@gmail.com 13700319475328460054304 13700319475328460054304 No Department of Anatomical Sciences, School of Medicine, Iran University of Medical Sciences, Tehran, Iran. Auob Rustamzadeh Auob2020rustamzade@gmail.com 13700319475328460054305 13700319475328460054305 No Cellular and Molecular Research Center, Research Institute for Prevention of Non-Communicable Disease, Qazvin University of Medical Sciences, Qazvin, Iran. Enam Alhagh Gorgich enam.gorgich@yahoo.com 13700319475328460054306 13700319475328460054306 No Department of Anatomy, School of Medicine, Iranshahr University of Medical Sciences, Iranshahr, Iran. Elham Seidkhani e.seidkhani@gmail.com 13700319475328460054307 13700319475328460054307 No Department of Anatomical Sciences, School of Medicine, Iran University of Medical Sciences, Tehran, Iran. Farnoosh Usefi f_useef@ymail.com 13700319475328460054308 0009-0009-6710-0974 No Department of Anatomical Sciences, School of Medicine, Iran University of Medical Sciences, Tehran, Iran. Mehdi Mehdizadeh m_mehdii12@ymail.com 13700319475328460054309 13700319475328460054309 No Department of Anatomical Sciences, School of Medicine, Iran University of Medical Sciences, Tehran, Iran. Fatemeh Moradi F7moradi@gmail.com 13700319475328460054310 13700319475328460054310 Yes Department of Anatomical Sciences, School of Medicine, Iran University of Medical Sciences, Tehran, Iran.