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What's new in HSCI research

A new high-throughput platform for discovering potential ALS drugs

Illustration of neurons in a lab dish.
HSCI researchers led by Clifford Woolf have developed a high-throughput platform for discovering drug targets to treat amyotrophic lateral sclerosis (ALS).
  • What they did: The researchers used stem cells derived from patients with ALS to create motor neurons, the cell type affected in ALS. They used imaging to measure whether drug candidates reduced the neurons’ hyperexcitability, or tendency to fire excessively.
  • What they found: After screening a library of 2,900 compounds, the researchers confirmed two known ALS drug targets and identified a new one.
  • Why it matters: A drug for one of the targets identified by the platform is currently under clinical development by QurAlis, a biotechnology company founded by Woolf and fellow HSCI faculty member Kevin Eggan. Additionally, their approach of using patient-derived cells to screen for drugs can be applied to many other diseases.

A detailed atlas of the developing brain

Microscopy image of the developing cerebral cortex.
HSCI researchers led by Paola Arlotta have created the first detailed atlas of a critical region of the developing mouse brain, focusing on the part of the cerebral cortex that is responsible for processing sensation from the body.
  • What they did: The researchers used multiple advanced genomic technologies to measure how gene activity and regulation changed over time, profiling almost every cell during every day of development to create a highly detailed picture of cortical development.
  • What they found: They were able to better understand how the brain develops, such as identifying when the cortex’s diversity of different neuron types is established.
  • Why it matters: Researchers have a new set of tools to explore how the brain is built and how it is affected during neurodevelopmental diseases.
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