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dc.contributor.authorMarchiò, Serenaen_US
dc.contributor.authorSidman, Richard Len_US
dc.contributor.authorArap, Wadihen_US
dc.contributor.authorPasqualini, Renataen_US
dc.date.accessioned2017-01-03T23:49:21Z
dc.date.issued2016en_US
dc.identifier.citationMarchiò, Serena, Richard L Sidman, Wadih Arap, and Renata Pasqualini. 2016. “Brain endothelial cell‐targeted gene therapy of neurovascular disorders.” EMBO Molecular Medicine 8 (6): 592-594. doi:10.15252/emmm.201606407. http://dx.doi.org/10.15252/emmm.201606407.en
dc.identifier.issn1757-4676en
dc.identifier.urihttp://nrs.harvard.edu/urn-3:HUL.InstRepos:29739078
dc.description.abstractNeurovascular 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.en
dc.language.isoen_USen
dc.publisherJohn Wiley and Sons Inc.en
dc.relation.isversionofdoi:10.15252/emmm.201606407en
dc.relation.hasversionhttp://www.ncbi.nlm.nih.gov/pmc/articles/PMC4888850/pdf/en
dash.licenseLAAen_US
dc.subjectNews & Viewsen
dc.subjectCardiovascular Systemen
dc.subjectGenetics, Gene Therapy & Genetic Diseaseen
dc.subjectNeuroscienceen
dc.titleBrain endothelial cell‐targeted gene therapy of neurovascular disordersen
dc.typeJournal Articleen_US
dc.description.versionVersion of Recorden
dc.relation.journalEMBO Molecular Medicineen
dash.depositing.authorSidman, Richard Len_US
dc.date.available2017-01-03T23:49:21Z
dc.identifier.doi10.15252/emmm.201606407*
dash.contributor.affiliatedSidman, Richard


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