Scientists create next generation of tools in battle against brain disease: The Armamentarium

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"In an exciting scientific first, researchers from around 29 universities and institutions across North America have teamed up to create a large, versatile, and effective arsenal of new biological tools that will play a critical role in the battle against brain disease. The tools are known as enhancer AAV vectors.

Scientists at the Allen Institute successfully created over 1000 of them, and each consist of:  

A harmless Adeno-associated virus (or AAV) that acts like a shuttle capable of transporting specially designed DNA into the cell.

A segment of DNA (the enhancer) that acts like an “activation switch” to mark or trigger a change in how the cell functions.

The cell-type specific targeting capability of these new genetic tools opens the door to targeted gene therapies that can correct genetic defects in specific cells that contribute to disease without affecting surrounding cells and adding unwanted side effects. Scientists now have new tools to help design the next generation of drugs to help treat brain disorders.

This type of cell-type targeted has never been demonstrated at this scale and effectiveness before. The findings were published in eight studies in the Cell Press family of journals (Cell, Neuron, Cell Reports Method, Cell Reports, Cell Genomics).  ...

Key Findings

1. The most notable and exciting finding suggests that many of these new tools will be safer and more effective for debilitating brain disorders. Researchers recently demonstrated the benefit of cell-type targeting in a recent breakthrough related to Dravet syndrome. 
2. Researchers successfully designed genomic tools for an incredible diversity of brain cell types, including for both cortex (Furlanis et al.; Ben-Simon et al.) and striatum (Hunker et al.) brain regions, as well as the spinal cord (Kussick et al.). 
3. The new tools were very effective at targeting specific brain cells and allowing scientists to successfully change cell activity and animal behavior in a predictable way. In one study, scientists successfully targeted and labeled a rare cell that regulates sleep, opening the door to new cell type-specific treatments for sleeping disorders. 

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Scientists create next generation of tools in battle against brain disease: The Armamentarium - Allen Institute "The findings contained in eight studies could lead to targeted gene therapies for brain disorders "