A new series of versatile and effective biological tools, which has a fundamental role in the fight against brain diseases, opens the door to directed gene therapies.
These tools called AAV Vectors Potentiators They are presented in a series of eight studies published by Cell and involving researchers from 29 universities and institutions headed by the Allen Institute (EU).
The ability of these new tools to specifically address a cell type opens the door to therapies that can correct genetic defects in specific cells that contribute to the disease without affecting the surrounding ones or add unwanted side effects, the institute said.
The new tools were “very effective when identifying specific brain cells and allowed scientists to modify cell activity and animal behavior predictably,” according to the same source.
Los AAV Vectors Potentiators They consist of a harmless adenoasociated virus (or AAV) that acts as a shuttle capable of transporting DNA specially designed inside the cell.
In addition, they have a DNA segment (the enhancer) that acts as an ‘activation switch’ to mark or trigger a change in the operation of the cell.
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They create a new generation of biological tools against brain diseases
Work is part of the Armamentarium for Precision Brain Cell Accessa project within the Brain initiative of the National Institutes of Health of the United States.
Diseases usually arise from failures in specific cell types, not throughout the body, thus epilepsy is a disease of specific nervous system.
Going to the appropriate cells, “in the appropriate and timely manner, is the future of precision brain medicine” and these tools bring us closer to that future, while expanding what is known today about cells and brain circuits, “said John Ngai, director of BRAIN.
Researchers have demonstrated the advantages of cell orientation in a recent advance related to Dravet syndrome.
In addition, in a study, they managed to locate and mark a rare cell that regulates sleep, which opens the door to new specific treatments for sleep disorders.
Likewise, they designed genomic tools for various types of brain cells, including the cerebral regions of the cortex, the striated body and the spinal cord.
With EFE information.
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