Extracellular Vesicles as Tools for Crossing the Blood–Brain Barrier to Treat Lysosomal Storage Diseases
Extracellular vesicles (EVs) are nanosized, membrane-bound structures that have emerged as promising tools for drug delivery, especially in the treatment of lysosomal storage disorders (LSDs) with central nervous system (CNS) involvement. This review highlights the unique properties of EVs, such as...
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| Format: | Article |
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MDPI AG
2025-01-01
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| Series: | Life |
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| Online Access: | https://www.mdpi.com/2075-1729/15/1/70 |
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| author | Giovanni Lerussi Verónica Villagrasa-Araya Marc Moltó-Abad Mireia del Toro Guillem Pintos-Morell Joaquin Seras-Franzoso Ibane Abasolo |
| author_facet | Giovanni Lerussi Verónica Villagrasa-Araya Marc Moltó-Abad Mireia del Toro Guillem Pintos-Morell Joaquin Seras-Franzoso Ibane Abasolo |
| author_sort | Giovanni Lerussi |
| collection | DOAJ |
| description | Extracellular vesicles (EVs) are nanosized, membrane-bound structures that have emerged as promising tools for drug delivery, especially in the treatment of lysosomal storage disorders (LSDs) with central nervous system (CNS) involvement. This review highlights the unique properties of EVs, such as their biocompatibility, capacity to cross the blood–brain barrier (BBB), and potential for therapeutic cargo loading, including that of enzymes and genetic material. Current therapies for LSDs, like enzyme replacement therapy (ERT), often fail to address neurological symptoms due to their inability to cross the BBB. EVs offer a viable alternative, allowing for targeted delivery to the CNS and improving therapeutic outcomes. We discuss recent advancements in the engineering and modification of EVs to enhance targeting, circulation time and cargo stability, and provide a detailed overview of their application in LSDs, such as Gaucher and Fabry diseases, and Sanfilippo syndrome. Despite their potential, challenges remain in scaling production, ensuring isolation purity, and meeting regulatory requirements. Future developments will focus on overcoming these barriers, paving the way for the clinical translation of EV-based therapies in LSDs and other CNS disorders. |
| format | Article |
| id | doaj-art-bc7b86df23b54fe5b2d396f377f0c7ea |
| institution | Kabale University |
| issn | 2075-1729 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Life |
| spelling | doaj-art-bc7b86df23b54fe5b2d396f377f0c7ea2025-01-24T13:38:40ZengMDPI AGLife2075-17292025-01-011517010.3390/life15010070Extracellular Vesicles as Tools for Crossing the Blood–Brain Barrier to Treat Lysosomal Storage DiseasesGiovanni Lerussi0Verónica Villagrasa-Araya1Marc Moltó-Abad2Mireia del Toro3Guillem Pintos-Morell4Joaquin Seras-Franzoso5Ibane Abasolo6Clinical Biochemistry, Drug Delivery & Therapy (CB-DDT), Vall d’Hebron Institute of Research (VHIR), 08035 Barcelona, SpainClinical Biochemistry, Drug Delivery & Therapy (CB-DDT), Vall d’Hebron Institute of Research (VHIR), 08035 Barcelona, SpainClinical Biochemistry, Drug Delivery & Therapy (CB-DDT), Vall d’Hebron Institute of Research (VHIR), 08035 Barcelona, SpainPediatric Neurology Unit, Hospital Universitari Vall d’Hebron and MetabERN, 08035 Barcelona, SpainClinical Biochemistry, Drug Delivery & Therapy (CB-DDT), Vall d’Hebron Institute of Research (VHIR), 08035 Barcelona, SpainClinical Biochemistry, Drug Delivery & Therapy (CB-DDT), Vall d’Hebron Institute of Research (VHIR), 08035 Barcelona, SpainNetworking Research Center on Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), 08035 Barcelona, SpainExtracellular vesicles (EVs) are nanosized, membrane-bound structures that have emerged as promising tools for drug delivery, especially in the treatment of lysosomal storage disorders (LSDs) with central nervous system (CNS) involvement. This review highlights the unique properties of EVs, such as their biocompatibility, capacity to cross the blood–brain barrier (BBB), and potential for therapeutic cargo loading, including that of enzymes and genetic material. Current therapies for LSDs, like enzyme replacement therapy (ERT), often fail to address neurological symptoms due to their inability to cross the BBB. EVs offer a viable alternative, allowing for targeted delivery to the CNS and improving therapeutic outcomes. We discuss recent advancements in the engineering and modification of EVs to enhance targeting, circulation time and cargo stability, and provide a detailed overview of their application in LSDs, such as Gaucher and Fabry diseases, and Sanfilippo syndrome. Despite their potential, challenges remain in scaling production, ensuring isolation purity, and meeting regulatory requirements. Future developments will focus on overcoming these barriers, paving the way for the clinical translation of EV-based therapies in LSDs and other CNS disorders.https://www.mdpi.com/2075-1729/15/1/70blood–brain barrierdrug deliveryenzyme replacement therapyexosomesextracellular vesicleslysosomal storage diseases |
| spellingShingle | Giovanni Lerussi Verónica Villagrasa-Araya Marc Moltó-Abad Mireia del Toro Guillem Pintos-Morell Joaquin Seras-Franzoso Ibane Abasolo Extracellular Vesicles as Tools for Crossing the Blood–Brain Barrier to Treat Lysosomal Storage Diseases Life blood–brain barrier drug delivery enzyme replacement therapy exosomes extracellular vesicles lysosomal storage diseases |
| title | Extracellular Vesicles as Tools for Crossing the Blood–Brain Barrier to Treat Lysosomal Storage Diseases |
| title_full | Extracellular Vesicles as Tools for Crossing the Blood–Brain Barrier to Treat Lysosomal Storage Diseases |
| title_fullStr | Extracellular Vesicles as Tools for Crossing the Blood–Brain Barrier to Treat Lysosomal Storage Diseases |
| title_full_unstemmed | Extracellular Vesicles as Tools for Crossing the Blood–Brain Barrier to Treat Lysosomal Storage Diseases |
| title_short | Extracellular Vesicles as Tools for Crossing the Blood–Brain Barrier to Treat Lysosomal Storage Diseases |
| title_sort | extracellular vesicles as tools for crossing the blood brain barrier to treat lysosomal storage diseases |
| topic | blood–brain barrier drug delivery enzyme replacement therapy exosomes extracellular vesicles lysosomal storage diseases |
| url | https://www.mdpi.com/2075-1729/15/1/70 |
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