Allen, Nick - Cardiff University - School of Biosciences

研究领域

Human IPSC models of neurodegenerative disease Neurodegenerative diseases are devastating for patients, families and society. Understanding the mechanistic basis of these diseases is fundamental to future drug development and therapeutic intervention. My group works on two diseases – Huntington’s (HD) and Alzheimer’s (AD). HD is an inherited genetic disorder caused by mutation in a single gene called huntingtin (Htt), in contrast AD is a multifactorial disease with a significant degree of risk gene heritability. A major research challenge is to model these diseases, to gain an understanding of disease mechanisms and to build platforms for future drug discovery and testing. To address this challenge my research group has focussed on the development of human cell models of disease. We take advantage of landmark ‘induced pluripotent stem cell technologies’ that ultimately enable disease models to be derived from patients – thereby directly integrating patient genetics into the models. My group has longstanding expertise in developmental neuroscience and stem cell biology. We harness this expertise to control stem cell differentiation to generate highly defined disease models that comprise the most disease–relevant cells for study. We collaborate extensively working within consortia for HD disease modeling (NINDS/CHDI HD iPS Consortium) and cell therapy (FP7 Repair HD) and for AD in the MRC/Dementia Platform UK (DPUK) stem cell network. For each program we provided expertise in stem cell differentiation, CRISPR-Cas9 genome editing and neurobiology. Our work on HD has led to novel biomarker discovery, provided progressive phenotyping, identified physiological deficits in the human neuronal models and led to engagement with industry to translate platforms for drug discovery.

近期论文

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