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We present an innovative drug delivery strategy centered on a peptide-targeted nanoliposomal formulation, designated as nanoGLA, engineered to selectively deliver a recombinant human GLA (rhGLA) to target tissues. In a Fabry mouse model, nanoGLA demonstrated improved efficacy, inducing a notable reduction in Gb3 deposits in contrast to non-nanoformulated GLA, even in the brain, highlighting the potential of the nanoGLA to address both systemic and cerebrovascular manifestations of Fabry disease. The EMA has granted the Orphan Drug Designation to this product, underscoring the potential clinical superiority of nanoGLA over authorized ERTs and encouraging to advance it toward clinical translation.<\/jats:p>","DOI":"10.1126\/sciadv.adq4738","type":"journal-article","created":{"date-parts":[[2024,12,13]],"date-time":"2024-12-13T19:00:22Z","timestamp":1734116422000},"update-policy":"https:\/\/doi.org\/10.34133\/aaas_crossmark","source":"Crossref","is-referenced-by-count":11,"title":["Targeted nanoliposomes to improve enzyme replacement therapy of Fabry disease"],"prefix":"10.1126","volume":"10","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2837-8107","authenticated-orcid":true,"given":"Judit","family":"Tomsen-Melero","sequence":"first","affiliation":[{"name":"Centro de Investigaci\u00f3n Biom\u00e9dica en Red de Bioingenier\u00eda, Biomateriales y Nanomedicina, Instituto de Salud Carlos III, Madrid, Spain."},{"name":"Institut de Ci\u00e8ncia de Materials de Barcelona, 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