All Outputs (3)

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

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

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.

A transcriptomic-based drug repositioning approach for the identification of novel muscle-specific therapies for spinal muscular atrophy (2022)
Thesis

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder (NMD) caused by depleted survival of motor neuron (SMN) levels and characterised by neuronal degeneration and progressive muscle atrophy. Although three approved SMN-depen... Read More about A transcriptomic-based drug repositioning approach for the identification of novel muscle-specific therapies for spinal muscular atrophy.