All Outputs (2)

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 (2023)
Journal Article
Selvakumaran, J., Ursu, S., Bowerman, M., Lu-Nguyen, N., Wood, M. J., Malerba, A., & Yáñez-Muñoz, R. J. (2023). 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. Biomedicines, 11(10), Article 2700. https://doi.org/10.3390/xxxxx

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 predictiv... Read More about 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.

Investigating the therapeutic potential of the glucocorticoid antagonist, mifepristone, for the treatment of spinal muscular atrophy (2023)
Thesis
Sutton, E. R. (2023). Investigating the therapeutic potential of the glucocorticoid antagonist, mifepristone, for the treatment of spinal muscular atrophy. (Thesis). Keele University

Spinal muscular atrophy (SMA) is a neuromuscular disease characterised by loss of alpha motor neurons and muscle atrophy. When untreated, mortality occurs within the first two years of life in the most severe cases. Extensive research has led to thre... Read More about Investigating the therapeutic potential of the glucocorticoid antagonist, mifepristone, for the treatment of spinal muscular atrophy.