A stem cell model to assess the passage of the blood-brain barrier

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British researchers have developed induced pluripotent stem cell (iPSC) lines to test the permeability of the blood-brain barrier to two therapeutic agents used in several neuromuscular diseases, including spinal muscular atrophy (SMA):

  • three iPSC lines obtained were differentiated into endothelial cells involved in brain microvascularisation,
  • a permeability index (endothelial electrical resistance or TEER) was defined, enabling the properties of several products to be compared,
  • the virus associated with adenovirus type 9 (AAV-9) passes the barrier better than AAV-8, whereas oligonucleotides do not.

This new tool will undoubtedly be very useful in the design of future gene therapies.

 

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. Selvakumaran J, Ursu S, Bowerman M et al. Biomedicines. 2023 Oct

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