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.
