An Induced Pluripotent Stem Cell-Derived Human Blood–Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides

An Induced Pluripotent Stem Cell-Derived Human Blood–Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides

The blood–brain barrier (BBB) is the specialised microvasculature system that shields the central nervous system (CNS) from potentially toxic agents. Attempts to develop therapeutic agents targeting the CNS have been hindered by the lack of predictive models of BBB crossing. In vitro models mimickin...

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Bibliographic Details
Main Authors: Jamuna Selvakumaran, Simona Ursu, Melissa Bowerman, Ngoc Lu-Nguyen, Matthew J. Wood, Alberto Malerba, Rafael J. Yáñez-Muñoz
Format: Article
Language:English
Published: MDPI AG 2023-10-01
Series:Biomedicines
Subjects:
human blood–brain barrier
brain microvascular endothelial cells
induced pluripotent stem cells
trans-endothelial electrical resistance
gene therapy
spinal muscular atrophy
Online Access:https://www.mdpi.com/2227-9059/11/10/2700

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