UCSD gene therapy preserves brain function by boosting protective protein rather than removing toxic TDP-43
By
Noah Lyons
Slow-proofed and worth the wait. Worth its weight in flour.
Summary
UC San Diego scientists have developed an experimental gene therapy called SynCav1 that takes a novel approach to fighting the toxic protein TDP-43, which is linked to ALS and Alzheimer's disease. Unlike most gene therapies that aim to remove toxic proteins, this therapy uses a modified harmless virus to deliver a gene that increases expression of a neuroprotective protein, helping preserve brain function despite the presence of the destructive protein. The research was led by Brian Head, a professor of anesthesiology at UCSD School of Medicine.
Key quotes
· 3 pulledThe new approach from scientists led by Brian Head, a professor of anesthesiology in the UCSD School of Medicine in La Jolla, uses a modified, harmless virus to systematically bring a gene therapy called SynCav1 to brain cells
The therapy increased expression of a neuroprotective protein to help preserve brain functions, despite the presence of a destructive protein linked to ALS and Alzheimer's
While most gene therapies target the removal of toxic proteins through injections to brain and spinal cord tissues, the new approach... uses a modified, harmless virus
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