Neurodegeneration impairs vital functions such as movement and memory. Alzheimer's disease (AD) constitutes the most common neurodegenerative diseases, manifesting upon aging. Similar disorders can manifest earlier in life, such as the lysosomal storage diseases, manifesting during childhood. Needless to say, these diseases cause widespread suffering. Unfortunately, there are generally speaking no curative therapies available, so novel therapeutic options are in high demand.

The last few years brought about techniques which should prove revolutionary in understanding neurodegeneration. While it was long impossible to obtain human nerve cells, the advent of human induced pluripotent stem cells (iPSCs) gives us access to cells of limited accessibility. Furthermore, gene editing tools (such as the gene scissors CRISPR/Cas9) provide an exciting opportunity to research diseases of interest. Upon combining iPSC technology and gene editing, scientists now have complete access to diseased human neurons, allowing us to research neurodegeneration in the optimal model system.

Combination of iPSC technology and gene editingi tools with global profiling approaches (the so-called "-omics" field, including proteomics, metabolomics, and transcriptomics) permits a broad, unbiased inquisition of pathology and revelation of druggable targets. As an avid pharmacologist and pathway biologist, I harness these techniques to develop new therapies for neurodegenerative disorders. I believe that now, as the tools required for such a venture are available, a cure for neurodegeneration is in sight - And I am thrilled to be part of it.