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...
Ausführliche Beschreibung
Gespeichert in:
| Autor*in: |
Marchiò, Serena [verfasserIn] Sidman, Richard L [verfasserIn] Arap, Wadih [verfasserIn] Pasqualini, Renata [verfasserIn] |
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| Format: |
E-Artikel |
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| Sprache: |
Englisch |
| Erschienen: |
2016 |
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| Anmerkung: |
© The Authors. Published under the terms of the CC BY 4.0 license 2016 |
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| Übergeordnetes Werk: |
Enthalten in: EMBO Molecular Medicine - Nature Publishing Group UK, 2023, 8(2016), 6 vom: 10. Mai, Seite 592-594 |
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| Übergeordnetes Werk: |
volume:8 ; year:2016 ; number:6 ; day:10 ; month:05 ; pages:592-594 |
| Links: |
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| DOI / URN: |
10.15252/emmm.201606407 |
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| Katalog-ID: |
SPR057908109 |
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| 520 | |a 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. | ||
| 520 | |a Graphical Abstract Commentary on the paper by Körbelin et al in this issue of EMBO Molecular Medicine, describing an engineered adeno‐associated virus (AAV) with exceptional tropism for the brain that restores a normal phenotype in a mouse incontinentia pigmenti model. | ||
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10.15252/emmm.201606407 doi (DE-627)SPR057908109 (SPR)emmm.201606407-e DE-627 ger DE-627 rakwb eng Marchiò, Serena verfasserin aut Brain endothelial cell‐targeted gene therapy of neurovascular disorders 2016 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © The Authors. Published under the terms of the CC BY 4.0 license 2016 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. Graphical Abstract Commentary on the paper by Körbelin et al in this issue of EMBO Molecular Medicine, describing an engineered adeno‐associated virus (AAV) with exceptional tropism for the brain that restores a normal phenotype in a mouse incontinentia pigmenti model. Sidman, Richard L verfasserin aut Arap, Wadih verfasserin (orcid)0000-0002-8686-4584 aut Pasqualini, Renata verfasserin (orcid)0000-0002-4155-9324 aut Enthalten in EMBO Molecular Medicine Nature Publishing Group UK, 2023 8(2016), 6 vom: 10. Mai, Seite 592-594 (DE-627)594772761 (DE-600)2485479-7 1757-4684 nnns volume:8 year:2016 number:6 day:10 month:05 pages:592-594 https://dx.doi.org/10.15252/emmm.201606407 X:SPRINGER Resolving-System kostenfrei Volltext SYSFLAG_0 GBV_SPRINGER GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_72 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_171 GBV_ILN_206 GBV_ILN_213 GBV_ILN_224 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_636 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2004 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2034 GBV_ILN_2037 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2093 GBV_ILN_2106 GBV_ILN_2108 GBV_ILN_2110 GBV_ILN_2111 GBV_ILN_2118 GBV_ILN_2122 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2144 GBV_ILN_2147 GBV_ILN_2148 GBV_ILN_2152 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2232 GBV_ILN_2336 GBV_ILN_2470 GBV_ILN_2507 GBV_ILN_2522 GBV_ILN_2548 GBV_ILN_4012 GBV_ILN_4029 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4046 GBV_ILN_4112 GBV_ILN_4116 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4155 GBV_ILN_4242 GBV_ILN_4246 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4328 GBV_ILN_4333 GBV_ILN_4334 GBV_ILN_4335 GBV_ILN_4336 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 AR 8 2016 6 10 05 592-594 |
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10.15252/emmm.201606407 doi (DE-627)SPR057908109 (SPR)emmm.201606407-e DE-627 ger DE-627 rakwb eng Marchiò, Serena verfasserin aut Brain endothelial cell‐targeted gene therapy of neurovascular disorders 2016 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © The Authors. Published under the terms of the CC BY 4.0 license 2016 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. Graphical Abstract Commentary on the paper by Körbelin et al in this issue of EMBO Molecular Medicine, describing an engineered adeno‐associated virus (AAV) with exceptional tropism for the brain that restores a normal phenotype in a mouse incontinentia pigmenti model. Sidman, Richard L verfasserin aut Arap, Wadih verfasserin (orcid)0000-0002-8686-4584 aut Pasqualini, Renata verfasserin (orcid)0000-0002-4155-9324 aut Enthalten in EMBO Molecular Medicine Nature Publishing Group UK, 2023 8(2016), 6 vom: 10. Mai, Seite 592-594 (DE-627)594772761 (DE-600)2485479-7 1757-4684 nnns volume:8 year:2016 number:6 day:10 month:05 pages:592-594 https://dx.doi.org/10.15252/emmm.201606407 X:SPRINGER Resolving-System kostenfrei Volltext SYSFLAG_0 GBV_SPRINGER GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_72 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_171 GBV_ILN_206 GBV_ILN_213 GBV_ILN_224 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_636 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2004 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2034 GBV_ILN_2037 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2093 GBV_ILN_2106 GBV_ILN_2108 GBV_ILN_2110 GBV_ILN_2111 GBV_ILN_2118 GBV_ILN_2122 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2144 GBV_ILN_2147 GBV_ILN_2148 GBV_ILN_2152 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2232 GBV_ILN_2336 GBV_ILN_2470 GBV_ILN_2507 GBV_ILN_2522 GBV_ILN_2548 GBV_ILN_4012 GBV_ILN_4029 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4046 GBV_ILN_4112 GBV_ILN_4116 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4155 GBV_ILN_4242 GBV_ILN_4246 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4328 GBV_ILN_4333 GBV_ILN_4334 GBV_ILN_4335 GBV_ILN_4336 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 AR 8 2016 6 10 05 592-594 |
| allfields_unstemmed |
10.15252/emmm.201606407 doi (DE-627)SPR057908109 (SPR)emmm.201606407-e DE-627 ger DE-627 rakwb eng Marchiò, Serena verfasserin aut Brain endothelial cell‐targeted gene therapy of neurovascular disorders 2016 Text txt rdacontent Computermedien c rdamedia Online-Ressource cr rdacarrier © The Authors. Published under the terms of the CC BY 4.0 license 2016 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. Graphical Abstract Commentary on the paper by Körbelin et al in this issue of EMBO Molecular Medicine, describing an engineered adeno‐associated virus (AAV) with exceptional tropism for the brain that restores a normal phenotype in a mouse incontinentia pigmenti model. Sidman, Richard L verfasserin aut Arap, Wadih verfasserin (orcid)0000-0002-8686-4584 aut Pasqualini, Renata verfasserin (orcid)0000-0002-4155-9324 aut Enthalten in EMBO Molecular Medicine Nature Publishing Group UK, 2023 8(2016), 6 vom: 10. Mai, Seite 592-594 (DE-627)594772761 (DE-600)2485479-7 1757-4684 nnns volume:8 year:2016 number:6 day:10 month:05 pages:592-594 https://dx.doi.org/10.15252/emmm.201606407 X:SPRINGER Resolving-System kostenfrei Volltext SYSFLAG_0 GBV_SPRINGER GBV_ILN_20 GBV_ILN_22 GBV_ILN_23 GBV_ILN_24 GBV_ILN_31 GBV_ILN_39 GBV_ILN_40 GBV_ILN_60 GBV_ILN_62 GBV_ILN_63 GBV_ILN_65 GBV_ILN_69 GBV_ILN_72 GBV_ILN_73 GBV_ILN_74 GBV_ILN_95 GBV_ILN_105 GBV_ILN_110 GBV_ILN_151 GBV_ILN_161 GBV_ILN_170 GBV_ILN_171 GBV_ILN_206 GBV_ILN_213 GBV_ILN_224 GBV_ILN_230 GBV_ILN_285 GBV_ILN_293 GBV_ILN_602 GBV_ILN_636 GBV_ILN_2001 GBV_ILN_2003 GBV_ILN_2004 GBV_ILN_2005 GBV_ILN_2006 GBV_ILN_2007 GBV_ILN_2009 GBV_ILN_2010 GBV_ILN_2011 GBV_ILN_2014 GBV_ILN_2015 GBV_ILN_2020 GBV_ILN_2021 GBV_ILN_2025 GBV_ILN_2026 GBV_ILN_2027 GBV_ILN_2034 GBV_ILN_2037 GBV_ILN_2038 GBV_ILN_2044 GBV_ILN_2048 GBV_ILN_2049 GBV_ILN_2050 GBV_ILN_2055 GBV_ILN_2056 GBV_ILN_2057 GBV_ILN_2059 GBV_ILN_2061 GBV_ILN_2064 GBV_ILN_2068 GBV_ILN_2088 GBV_ILN_2093 GBV_ILN_2106 GBV_ILN_2108 GBV_ILN_2110 GBV_ILN_2111 GBV_ILN_2118 GBV_ILN_2122 GBV_ILN_2129 GBV_ILN_2143 GBV_ILN_2144 GBV_ILN_2147 GBV_ILN_2148 GBV_ILN_2152 GBV_ILN_2153 GBV_ILN_2190 GBV_ILN_2232 GBV_ILN_2336 GBV_ILN_2470 GBV_ILN_2507 GBV_ILN_2522 GBV_ILN_2548 GBV_ILN_4012 GBV_ILN_4029 GBV_ILN_4035 GBV_ILN_4037 GBV_ILN_4046 GBV_ILN_4112 GBV_ILN_4116 GBV_ILN_4125 GBV_ILN_4126 GBV_ILN_4155 GBV_ILN_4242 GBV_ILN_4246 GBV_ILN_4249 GBV_ILN_4251 GBV_ILN_4305 GBV_ILN_4306 GBV_ILN_4307 GBV_ILN_4313 GBV_ILN_4322 GBV_ILN_4323 GBV_ILN_4324 GBV_ILN_4325 GBV_ILN_4326 GBV_ILN_4328 GBV_ILN_4333 GBV_ILN_4334 GBV_ILN_4335 GBV_ILN_4336 GBV_ILN_4338 GBV_ILN_4367 GBV_ILN_4393 GBV_ILN_4700 AR 8 2016 6 10 05 592-594 |
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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. Graphical Abstract Commentary on the paper by Körbelin et al in this issue of EMBO Molecular Medicine, describing an engineered adeno‐associated virus (AAV) with exceptional tropism for the brain that restores a normal phenotype in a mouse incontinentia pigmenti model. © The Authors. Published under the terms of the CC BY 4.0 license 2016 |
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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. Graphical Abstract Commentary on the paper by Körbelin et al in this issue of EMBO Molecular Medicine, describing an engineered adeno‐associated virus (AAV) with exceptional tropism for the brain that restores a normal phenotype in a mouse incontinentia pigmenti model. © The Authors. Published under the terms of the CC BY 4.0 license 2016 |
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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. Graphical Abstract Commentary on the paper by Körbelin et al in this issue of EMBO Molecular Medicine, describing an engineered adeno‐associated virus (AAV) with exceptional tropism for the brain that restores a normal phenotype in a mouse incontinentia pigmenti model. © The Authors. Published under the terms of the CC BY 4.0 license 2016 |
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| score |
7.398796 |