A patient-derived motor platform for diagnostics and drug discovery in neuromuscular diseases : a dissertation presented in fulfilment of the requirements for the degree of Doctor of Philosophy in Biochemistry, Massey University, Manawatū, New Zealand. EMBARGOED to 7 May 2027.
| dc.confidential | Embargo : Yes | |
| dc.contributor.advisor | Stowell, Kathryn | |
| dc.contributor.author | Burling, Sophie | |
| dc.date.accessioned | 2025-06-02T21:41:38Z | |
| dc.date.available | 2025-06-02T21:41:38Z | |
| dc.date.issued | 2025-01-29 | |
| dc.description.abstract | This research developed patient-derived multicellular disease models to advance diagnosis and drug discovery for neuromuscular and neurodegenerative diseases, such as malignant hyperthermia and motor neurone disease. A biobank of stem cells reprogrammed from patients facilitated the creation of “mini organoids” that replicate disease features. A muscle-on-chip platform utilised a microfluidic system for precise drug delivery, demonstrating potential to replace invasive diagnostics, and in ALS models, a novel repurposed drug effectively reduced key disease markers. The outcome is a scalable, patient-specific platform designed to enhance disease understanding, improve diagnostics, and expedite therapies for incurable and complex diseases. | |
| dc.identifier.uri | https://mro.massey.ac.nz/handle/10179/72980 | |
| dc.publisher | Massey University | |
| dc.publisher | Embargoed to 7 May 2027 | |
| dc.rights | © The Author | |
| dc.subject | stem cells, iPSC, muscle, amyotrophic lateral sclerosis, malignant hyperthermia, disease modelling, tissue engineering, drug discovery, diagnostics, neurodegenerative, neuromuscular disease, multicellular, microfluidics, organ-on-chip | |
| dc.subject | Neuromuscular diseases | |
| dc.subject | Pathogenesis | |
| dc.subject | Induced pluripotent stem cells | |
| dc.subject | Biochips | |
| dc.subject | Neuropharmacology | |
| dc.subject.anzsrc | 3101 Biochemistry and cell biology | |
| dc.subject.anzsrc | 320905 Neurology and neuromuscular diseases | |
| dc.title | A patient-derived motor platform for diagnostics and drug discovery in neuromuscular diseases : a dissertation presented in fulfilment of the requirements for the degree of Doctor of Philosophy in Biochemistry, Massey University, Manawatū, New Zealand. EMBARGOED to 7 May 2027. | |
| thesis.degree.discipline | Biochemistry | |
| thesis.degree.name | Doctor of Philosophy (Ph.D.) | |
| thesis.description.doctoral-citation-abridged | This research developed patient-derived multicellular disease models to enhance diagnostics and drug discovery for neuromuscular and neurodegenerative disorders, including motor neurone disease (MND). A biobank of patient stem cells was used to create patient-specific models, and a novel drug for MND was tested and showed promise. This has the potential to advance treatment options for these incurable diseases. | |
| thesis.description.doctoral-citation-long | This research developed patient-derived multicellular disease models to advance diagnosis and drug discovery for neuromuscular and neurodegenerative diseases, such as malignant hyperthermia and motor neurone disease. A biobank of stem cells reprogrammed from patients facilitated the creation of “mini organoids” that replicate disease features. A muscle-on-chip platform utilised a microfluidic system for precise drug delivery, demonstrating potential to replace invasive diagnostics, and in ALS models, a novel repurposed drug effectively reduced key disease markers. The outcome is a scalable, patient-specific platform designed to enhance disease understanding, improve diagnostics, and expedite therapies for incurable and complex diseases. | |
| thesis.description.name-pronounciation | SO FEE BUR LING |