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What's new in HSCI research

Engineered stem cells used to treat aggressive brain cancer

Glioblastomas (GBMs) are highly aggressive cancerous tumors of the brain and spinal cord. Brain cancers like GBM are challenging to treat, and more than 90 percent of GBM tumors return after being surgically removed, despite surgery and subsequent chemo- and radiation therapy. HSCI Principal Faculty member Khalid Shah developed a new strategy to treat these aggressive brain cancers.
  • What they did: The researchers devised a novel therapeutic strategy for treating GBMs post-surgery by identifying targets on the cancer surface and using stem cells taken from healthy donors engineered to attack GBM-specific tumor cells.
  • What they found: This strategy, which used biodegradable, gel-encapsulated, engineered stem cells, demonstrated profound efficacy in preclinical models of GBM, with 100 percent of mice living over 90 days after treatment.
  • Why it matters: This study establishes a foundation towards a clinical trial of engineered stem cells in patients with primary and recurrent GBM.

Scientists discover a dual function of a key protein Parkinson’s Disease protein

One of the hallmarks of Parkinson’s disease (PD) is the accumulation in the brain of a protein known as alpha-synuclein. But alpha-synuclein’s function is not well understood. New research from HSCI principal faculty member Vikram Khurana, MD, PhD, uncovered a new function for the protein with relevance for PD and related conditions.
  • What they did: The researchers studied neurons with alpha-synuclein gene mutations and post-mortem brain tissue samples from PD patients to learn more about the protein’s function.
  • What they found: The team discovered that alpha-synuclein is involved in the expression of certain genes. It appears the protein acts as a “toggle switch” that regulates two very distinct functions: transport of vesicles (previously known) and gene expression (a new discovery). In disease states, the balance is broken.
  • Why it matters: Patients who had an accumulation of mutated genes appeared to be at higher risk for PD. This discovery provides new insights for treatment of the disease.

Studying zebrafish social patterns reveals clues about psychiatric disease in humans

Studying zebrafish social patterns reveals clues about psychiatric disease in humans
Psychiatric diseases, like autism and schizophrenia, are marked often by deficits in social interaction. To learn more about the connection between genes and social behavior, internationally recognized geneticist and HSCI Principal Faculty member Mark Fishman uses zebrafish to understand how genetic changes in individuals affect how groups form and behave. The hope is that by understanding the genetics of social behavior in an animal that is genetically related to humans but much more easily studied, researchers could ultimately find new therapeutics for disorders like autism and schizophrenia.
  • What they did: The researchers introduced several different types of mutations into the genes of zebrafish – those known to be associated with autism, schizophrenia, and addiction - and studied their individual and collective behavior.
  • What they found: Their studies revealed that the mutations introduced into a single zebrafish led to different behaviors within the group; some mutations caused the fish to scatter; others led them to collectively huddle.
  • Why it matters: The results showed that making certain genetic modifications predicted how an animal will interact in a group. The research team hopes this discovery may lead to a better understanding of the synaptic circuitry that underlies autism, schizophrenia, and other diseases and ultimately to new therapies in the field of psychiatry.
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