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Gene therapy offers important therapeutic potential for brain disorders; however, its clinical translation is hindered by critical obstacles, particularly the blood\u2013brain barrier, which limits systemic delivery of therapeutic genes to the brain, an issue especially pronounced in primates. Here, we evaluated the use of low\u2010intensity focused ultrasound combined with intravenously administered microbubbles to transiently open the blood\u2013brain barrier in the cerebellum of macaque monkeys, thus enabling targeted delivery of adeno\u2010associated virus\u2010based gene therapy vectors. Two vector types (scAAV9\u2010CBA\u2010GFP and ssAAV9\u2010CMV\u2010mCherry) were administered systemically, and transgene expression was analyzed to assess delivery efficiency and cell\u2010type distribution. We achieved successful, non\u2010invasive delivery of both vector types to the cerebellum with high spatial precision. Administration of ssAAV9\u2010CMV\u2010mCherry vector resulted in robust transduction of virtually all neurons within the targeted deep cerebellar nuclei. These findings provide a promising and translationally relevant strategy for developing gene\u2010delivery approaches for cerebellar and other neurodegenerative disorders and represent a step forward in advancing the use of focused ultrasound to achieve efficient and less invasive gene delivery to the brain.<\/jats:p>","DOI":"10.1002\/advs.75307","type":"journal-article","created":{"date-parts":[[2026,4,20]],"date-time":"2026-04-20T10:31:38Z","timestamp":1776681098000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Noninvasive Focal Gene Delivery into the Cerebellum of Non\u2010Human Primates using Focused Ultrasound"],"prefix":"10.1002","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2741-3054","authenticated-orcid":false,"given":"Noelia","family":"Esteban\u2010Garc\u00eda","sequence":"first","affiliation":[{"name":"HM CINAC (Centro Integral de Neurociencias Abarca Campal) Hospital Universitario 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