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STAT+: Scientists debut map of basal ganglia, a key brain region involved in an array of diseases

By · Via STAT Health · Published by Everything Blog

In short

The extensive effort mapped the diverse cell varieties across the basal ganglia while linking distinct types to depression, bipolar disorder, and post-traumatic stress disorder. Scientists also uncovered a previously unknown neuron that appears to play a functional role in schizophrenia. In experimental tests involving mouse models of Parkinson’s disease, a gene therapy focused strictly on a subset of these cells matched the efficacy of broad treatments while demonstrating a superior safety profile.

Key points

  • Targeted gene therapies can match the efficacy of broad interventions while providing sig…: Targeted gene therapies can match the efficacy of broad interventions while providing significantly safer outcomes.
  • Detailed biological cellular catalogs reveal novel mechanisms that underpin complex psych…: Detailed biological cellular catalogs reveal novel mechanisms that underpin complex psychiatric and neurological conditions.
  • Mapping brain cell types allows future clinical treatments to be engineered with much gre…: Mapping brain cell types allows future clinical treatments to be engineered with much greater biological precision.

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A massive scientific collaboration meant to unravel the mysteries of the human brain has produced the most detailed map yet of the basal ganglia, a region closely associated with a wide array of psychiatric and neurologic disorders.

Researchers profiled more than 25 million cells, generating a deluge of data that allowed them to generate a “parts list” of what cells are present in the basal ganglia and how they’re arranged. In the process scientists discovered a new type of neuron that appears to play a role in schizophrenia. They also identified cell types linked to depression, bipolar disorder, and post-traumatic stress disorder.

Demonstrating the value of the work, the scientists produced preliminary evidence that they can use their more detailed understanding of brain cell types to deliver therapies with increased precision. Authors working in mouse models of Parkinson’s disease showed that a gene therapy designed to work in a subset of basal ganglia cells was as effective as a blanket approach; it was also safer.

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