Uses for humanised mouse models in precision medicine for neurodegenerative disease.
Remya R NairSilvia CorrochanoSamanta GascoCharlotte TibbitDavid ThompsonCheryl MaduroZeinab AliPietro FrattaAbraham Acevedo ArozenaThomas J CunninghamElizabeth M C FisherPublished in: Mammalian genome : official journal of the International Mammalian Genome Society (2019)
Neurodegenerative disease encompasses a wide range of disorders afflicting the central and peripheral nervous systems and is a major unmet biomedical need of our time. There are very limited treatments, and no cures, for most of these diseases, including Alzheimer's Disease, Parkinson's Disease, Huntington Disease, and Motor Neuron Diseases. Mouse and other animal models provide hope by analysing them to understand pathogenic mechanisms, to identify drug targets, and to develop gene therapies and stem cell therapies. However, despite many decades of research, virtually no new treatments have reached the clinic. Increasingly, it is apparent that human heterogeneity within clinically defined neurodegenerative disorders, and between patients with the same genetic mutations, significantly impacts disease presentation and, potentially, therapeutic efficacy. Therefore, stratifying patients according to genetics, lifestyle, disease presentation, ethnicity, and other parameters may hold the key to bringing effective therapies from the bench to the clinic. Here, we discuss genetic and cellular humanised mouse models, and how they help in defining the genetic and environmental parameters associated with neurodegenerative disease, and so help in developing effective precision medicine strategies for future healthcare.
Keyphrases
- stem cells
- healthcare
- primary care
- mouse model
- emergency department
- magnetic resonance imaging
- genome wide
- physical activity
- metabolic syndrome
- gene expression
- mesenchymal stem cells
- climate change
- weight loss
- cognitive decline
- mild cognitive impairment
- electronic health record
- case report
- transcription factor
- drug induced