Projects per year
Abstract
Gene therapy critically relies on vectors that combine high transduction efficiency with a high degree of target specificity and that can be administered through a safe intravenous route. The lack of suitable vectors, especially for gene therapy of brain disorders, represents a major obstacle. Therefore, we applied an in vivo screening system of random ligand libraries displayed on adeno-associated viral capsids to select brain-targeted vectors for the treatment of neurovascular diseases. We identified a capsid variant showing an unprecedented degree of specificity and long-lasting transduction efficiency for brain microvasculature endothelial cells as the primary target of selection. A therapeutic vector based on this selected viral capsid was used to markedly attenuate the severe cerebrovascular pathology of mice with incontinentia pigmenti after a single intravenous injection. Furthermore, the versatility of this selection system will make it possible to select ligands for additional in vivo targets without requiring previous identification of potential target-specific receptors.
Original language | English |
---|---|
Journal | EMBO Molecular Medicine |
Volume | 8 |
Issue number | 6 |
Pages (from-to) | 609-625 |
Number of pages | 17 |
ISSN | 1757-4676 |
DOIs | |
Publication status | Published - 01.06.2016 |
Research Areas and Centers
- Academic Focus: Center for Brain, Behavior and Metabolism (CBBM)
Fingerprint
Dive into the research topics of 'A brain microvasculature endothelial cell-specific viral vector with the potential to treat neurovascular and neurological diseases'. Together they form a unique fingerprint.Projects
- 1 Finished
-
Novel brain endothelial-targeted gene therapy vectors to treat neuroinflammatory disease
Schwaninger, M. & Trepel, M.
01.08.14 → 31.07.17
Project: DFG Projects › DFG Individual Projects