Publications by authors named "Simon McDowall"
Article Synopsis
- Researchers are investigating new ways to replace or regenerate lost neurons in neurodegenerative diseases, specifically through direct reprogramming of astrocytes into neurons by manipulating a protein called PTBP1.
- Some studies claim that this reprogramming leads to functional neurons and better disease outcomes in animal models, while others challenge these findings, questioning the effectiveness of PTBP1 suppression for this purpose.
- The review highlights recent advancements in regenerative treatments like stem cell transplants for conditions such as Parkinson's and Alzheimer's, suggesting that differences in astrocyte types may explain the mixed results in research regarding their conversion to neurons.*
Antisense oligonucleotides and their applications in rare neurological diseases.
Front Neurosci
September 2024
Rare diseases affect almost 500 million people globally, predominantly impacting children and often leading to significantly impaired quality of life and high treatment costs. While significant contributions have been made to develop effective treatments for those with rare diseases, more rapid drug discovery strategies are needed. Therapeutic antisense oligonucleotides can modulate target gene expression with high specificity through various mechanisms determined by base sequences and chemical modifications; and have shown efficacy in clinical trials for a few rare neurological conditions.
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