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.
adeno-associated virus vectors
antisense oligonucleotides
brain microvascular endothelial cells
gene therapy
human blood–brain barrier
induced pluripotent stem cells
phosphorodiamidate morpholino oligomers
spinal muscular atrophy
trans-endothelial electrical resistance
Journal
Biomedicines
ISSN: 2227-9059
Titre abrégé: Biomedicines
Pays: Switzerland
ID NLM: 101691304
Informations de publication
Date de publication:
04 Oct 2023
04 Oct 2023
Historique:
received:
24
08
2023
revised:
26
09
2023
accepted:
28
09
2023
medline:
28
10
2023
pubmed:
28
10
2023
entrez:
28
10
2023
Statut:
epublish
Résumé
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 mimicking the human BBB are of great interest, and advances in induced pluripotent stem cell (iPSC) technologies and the availability of reproducible differentiation protocols have facilitated progress. In this study, we present the efficient differentiation of three different wild-type iPSC lines into brain microvascular endothelial cells (BMECs). Once differentiated, cells displayed several features of BMECs and exhibited significant barrier tightness as measured by trans-endothelial electrical resistance (TEER), ranging from 1500 to >6000 Ωcm
Identifiants
pubmed: 37893074
pii: biomedicines11102700
doi: 10.3390/biomedicines11102700
pmc: PMC10604610
pii:
doi:
Types de publication
Journal Article
Langues
eng
Subventions
Organisme : Biotechnology and Biological Sciences Research Council
ID : N/A
Pays : United Kingdom
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