دورية أكاديمية
Nanomedicines to treat rare neurological disorders: The case of Krabbe disease.
| العنوان: | Nanomedicines to treat rare neurological disorders: The case of Krabbe disease. |
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| المؤلفون: | Moore TL; Laboratory of Nanotechnology for Precision Medicine, Istituto Italiano di Tecnologia, Genoa 16163, GE, Italy. Electronic address: thomas.moore@iit.it., Pannuzzo G; Department of Biomedical and Biotechnological Sciences, Università di Catania, Catania 95123, CT, Italy., Costabile G; Laboratory of Nanotechnology for Precision Medicine, Istituto Italiano di Tecnologia, Genoa 16163, GE, Italy; Department of Pharmacy, Università degli Studi di Napoli Federico II, Naples 80131, NA, Italy., Palange AL; Laboratory of Nanotechnology for Precision Medicine, Istituto Italiano di Tecnologia, Genoa 16163, GE, Italy., Spanò R; Laboratory of Nanotechnology for Precision Medicine, Istituto Italiano di Tecnologia, Genoa 16163, GE, Italy., Ferreira M; Laboratory of Nanotechnology for Precision Medicine, Istituto Italiano di Tecnologia, Genoa 16163, GE, Italy., Graziano ACE; Department of Biomedical and Biotechnological Sciences, Università di Catania, Catania 95123, CT, Italy; Facolta di Medicina e Chirurgia, Università degli Studi di Enna 'Kore', Enna 94100, EN, Italy., Decuzzi P; Laboratory of Nanotechnology for Precision Medicine, Istituto Italiano di Tecnologia, Genoa 16163, GE, Italy., Cardile V; Department of Biomedical and Biotechnological Sciences, Università di Catania, Catania 95123, CT, Italy. Electronic address: cardile@unict.it. |
| المصدر: | Advanced drug delivery reviews [Adv Drug Deliv Rev] 2023 Dec; Vol. 203, pp. 115132. Date of Electronic Publication: 2023 Oct 31. |
| نوع المنشور: | Journal Article; Review; Research Support, Non-U.S. Gov't |
| اللغة: | English |
| بيانات الدورية: | Publisher: Elsevier Science Publishers, B.V Country of Publication: Netherlands NLM ID: 8710523 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1872-8294 (Electronic) Linking ISSN: 0169409X NLM ISO Abbreviation: Adv Drug Deliv Rev Subsets: MEDLINE |
| أسماء مطبوعة: | Original Publication: Amsterdam : Elsevier Science Publishers, B.V., c1987- |
| مواضيع طبية MeSH: | Leukodystrophy, Globoid Cell*/drug therapy , Leukodystrophy, Globoid Cell*/genetics , Lysosomal Storage Diseases*/drug therapy, Humans ; Galactosylceramidase/genetics ; Galactosylceramidase/metabolism ; Nanomedicine ; Brain/metabolism ; Blood-Brain Barrier/metabolism |
| مستخلص: | The brain remains one of the most challenging therapeutic targets due to the low and selective permeability of the blood-brain barrier and complex architecture of the brain tissue. Nanomedicines, despite their relatively large size compared to small molecules and nucleic acids, are being heavily investigated as vehicles to delivery therapeutics into the brain. Here we elaborate on how nanomedicines may be used to treat rare neurodevelopmental disorders, using Krabbe disease (globoid cell leukodystrophy) to frame the discussion. As a monogenetic disorder and lysosomal storage disease affecting the nervous system, the lessons learned from examining nanoparticle delivery to the brain in the context of Krabbe disease can have a broader impact on the treatment of various other neurodevelopmental and neurodegenerative disorders. In this review, we introduce the epidemiology and genetic basis of Krabbe disease, discuss current in vitro and in vivo models of the disease, as well as current therapeutic approaches either approved or at different stage of clinical developments. We then elaborate on challenges in particle delivery to the brain, with a specific emphasis on methods to transport nanomedicines across the blood-brain barrier. We highlight nanoparticles for delivering therapeutics for the treatment of lysosomal storage diseases, classified by the therapeutic payload, including gene therapy, enzyme replacement therapy, and small molecule delivery. Finally, we provide some useful hints on the design of nanomedicines for the treatment of rare neurological disorders. Competing Interests: Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper. (Copyright © 2023 The Author(s). Published by Elsevier B.V. All rights reserved.) |
| فهرسة مساهمة: | Keywords: Blood–brain barrier; Drug delivery; Globoid cell leukodystrophy; Krabbe disease; Lysosomal storage disease; Nanomedicine |
| المشرفين على المادة: | EC 3.2.1.46 (Galactosylceramidase) |
| تواريخ الأحداث: | Date Created: 20231102 Date Completed: 20231211 Latest Revision: 20231211 |
| رمز التحديث: | 20231215 |
| DOI: | 10.1016/j.addr.2023.115132 |
| PMID: | 37918668 |
| قاعدة البيانات: | MEDLINE |
Full Text Finder | تدمد: | 1872-8294 |
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| DOI: | 10.1016/j.addr.2023.115132 |