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Longevity Briefs: NIH Launch the Worlds Largest iPSC Genome-engineering Project to tackle Alzheimer’s and Dementia

Posted on 13 May 2021

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Longevity briefs provides a short summary of novel research in biology, medicine, or biotechnology that caught the attention of our researchers in Oxford, due to its potential to improve our health, wellbeing, and longevity.

Why is this research important: Alzheimer’s disease is the most common form of dementia, a neurological condition which sees the decline of cognitive ability to a senile state, eventually resulting in death. Over the next 20 years, the number of Alzheimer’s sufferers is set to double, to well over a million, in the UK.

Despite it being such a common disease we still know relatively little about it. This lack of understanding has limited our ability to produce effective therapies.

What did the researchers do: In a bid to tackle this problem, Dr. Michael E. Ward, principal investigator at NIH’s National Institute of Neurological Disorders and Stroke (NINDS), and Dr. Mark Cookson, principal investigator at the NIH’s National Institute on Aging (NIA), have launched the iPSC Neurodegenerative Disease Initiative (iNDI).

The iNDL is a project that aims to help scientists further develop their understanding of how genetic mutations may contribute to degenerative diseases of the brain, including Alzheimer’s.

iNDI relies on induced pluripotent stem cells (iPSCs), but, what exactly are they?

Pluripotent stem cells are cells with the potential to differentiate, or turn, into almost any other cell type in the body. Induced pluripotent stem cells are those which have been generated from an already differentiated body cell, such as a blood or skin cell. This is achieved by molecules known as yamanaka factors, which drive the epigenetic reprogramming of the original cell. As they are derived from the participants’ cells, they allow the study of the disorder in disease-relevant cell types with the patient’s genetic background, a major advantage.

iPSCs. Source: metamorworks

Key takeaway(s) from this research: The iNDI will be the largest iPSC genome-engineering project to date, with researchers aiming to create a new set of induced pluripotent stem cell lines, each with 1 out of over 100 disease-causing mutations.

Overview of iNDI. Source: Tackling neurodegenerative diseases with genomic engineering: A new stem cell initiative from the NIH

Scientists from around the globe will be given access to samples of each cell line, along with any relevant data resulting from future experiments. The team hopes this genetic database will accelerate the development of promising treatments for millions of people who suffer from these neurodegenerative disorders.

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