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Mutation of p53\u2010induced protein phosphatase 1 (PPM1D) in DIPG cells promotes tumor cell proliferation, and inhibition of PPM1D expression in DIPG cells with PPM1D mutation effectively reduces the proliferation activity of tumor cells. Panobinostat effectively kills DIPG tumor cells, but its systemic toxicity and low blood\u2013brain barrier (BBB) permeability limits its application. In this paper, a nano drug delivery system based on functionalized macrophage exosomes with panobinostat and PPM1D\u2010siRNA for targeted therapy of DIPG with PPM1D mutation is prepared. The nano drug delivery system has higher drug delivery efficiency and better therapeutic effect than free drugs. In vivo and in vitro experimental results show that the nano drug delivery system can deliver panobinostat and siRNA across the BBB and achieve a targeted killing effect of DIPG tumor cells, resulting in the prolonged survival of orthotopic DIPG mice. This study provides new ideas for the delivery of small molecule drugs and gene drugs for DIPG therapy.<\/jats:p>","DOI":"10.1002\/advs.202200353","type":"journal-article","created":{"date-parts":[[2022,5,18]],"date-time":"2022-05-18T23:59:55Z","timestamp":1652918395000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":89,"title":["Functionalized Macrophage Exosomes with Panobinostat and PPM1D\u2010siRNA for Diffuse Intrinsic Pontine Gliomas Therapy"],"prefix":"10.1002","volume":"9","author":[{"given":"Shaobo","family":"Shan","sequence":"first","affiliation":[{"name":"Key Laboratory for Biomechanics and Mechanobiology of Ministry of Education Beijing Advanced Innovation Center for Biomedical Engineering School of Biological Science and Medical Engineering &amp; School of Engineering Medicine &amp; Shenzhen Institute of Beihang University Beihang University  Beijing 100083 P. R. 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