Brain endothelial cell‐targeted gene therapy of neurovascular disorders
Abstract Neurovascular disorders are difficult to treat due to the blood–brain barrier (BBB), which prevents delivery of most drugs from the blood into the brain. Gene therapy can potentially overcome this barrier, but classical vectors are neither efficient nor specific enough to provide long‐lasti...
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| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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Springer Nature
2016-05-01
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| Series: | EMBO Molecular Medicine |
| Online Access: | https://doi.org/10.15252/emmm.201606407 |
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| _version_ | 1849761556736245760 |
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| author | Serena Marchiò Richard L Sidman Wadih Arap Renata Pasqualini |
| author_facet | Serena Marchiò Richard L Sidman Wadih Arap Renata Pasqualini |
| author_sort | Serena Marchiò |
| collection | DOAJ |
| description | Abstract Neurovascular disorders are difficult to treat due to the blood–brain barrier (BBB), which prevents delivery of most drugs from the blood into the brain. Gene therapy can potentially overcome this barrier, but classical vectors are neither efficient nor specific enough to provide long‐lasting treatment or avoid off‐target effects. In this issue of EMBO Molecular Medicine, Körbelin et al (2016) describe an engineered adeno‐associated virus (AAV) with exceptional tropism for the brain that restores a normal phenotype in a mouse model of incontinentia pigmenti (IP), a severe human genetic disorder of brain vasculature. |
| format | Article |
| id | doaj-art-ad9b36cec15a47879f662fbb001e7f4a |
| institution | DOAJ |
| issn | 1757-4676 1757-4684 |
| language | English |
| publishDate | 2016-05-01 |
| publisher | Springer Nature |
| record_format | Article |
| series | EMBO Molecular Medicine |
| spelling | doaj-art-ad9b36cec15a47879f662fbb001e7f4a2025-08-20T03:06:00ZengSpringer NatureEMBO Molecular Medicine1757-46761757-46842016-05-018659259410.15252/emmm.201606407Brain endothelial cell‐targeted gene therapy of neurovascular disordersSerena Marchiò0Richard L Sidman1Wadih Arap2Renata Pasqualini3University of New Mexico Comprehensive Cancer CenterDepartment of Neurology, Beth Israel Deaconess Medical Center, Harvard Medical SchoolDivision of Hematology and Oncology, Department of Internal Medicine, University of New Mexico School of MedicineUniversity of New Mexico Comprehensive Cancer CenterAbstract Neurovascular disorders are difficult to treat due to the blood–brain barrier (BBB), which prevents delivery of most drugs from the blood into the brain. Gene therapy can potentially overcome this barrier, but classical vectors are neither efficient nor specific enough to provide long‐lasting treatment or avoid off‐target effects. In this issue of EMBO Molecular Medicine, Körbelin et al (2016) describe an engineered adeno‐associated virus (AAV) with exceptional tropism for the brain that restores a normal phenotype in a mouse model of incontinentia pigmenti (IP), a severe human genetic disorder of brain vasculature.https://doi.org/10.15252/emmm.201606407 |
| spellingShingle | Serena Marchiò Richard L Sidman Wadih Arap Renata Pasqualini Brain endothelial cell‐targeted gene therapy of neurovascular disorders EMBO Molecular Medicine |
| title | Brain endothelial cell‐targeted gene therapy of neurovascular disorders |
| title_full | Brain endothelial cell‐targeted gene therapy of neurovascular disorders |
| title_fullStr | Brain endothelial cell‐targeted gene therapy of neurovascular disorders |
| title_full_unstemmed | Brain endothelial cell‐targeted gene therapy of neurovascular disorders |
| title_short | Brain endothelial cell‐targeted gene therapy of neurovascular disorders |
| title_sort | brain endothelial cell targeted gene therapy of neurovascular disorders |
| url | https://doi.org/10.15252/emmm.201606407 |
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