News Health/Medical Gene Therapy Breakthrough: Engineered Virus Crosses Blood-Brain Barrier
Gene delivery

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Gene Therapy Breakthrough: Engineered Virus Crosses Blood-Brain Barrier

May 16, 2024

Researchers have achieved a significant milestone in gene therapy by engineering a delivery vehicle that efficiently crosses the blood-brain barrier in mice. This breakthrough holds promise for safer and more effective treatments for various brain diseases.

Key Facts

  • A new gene delivery vehicle uses a human protein to efficiently cross the blood-brain barrier.
  • This breakthrough could lead to safer and more effective gene therapies for various brain diseases.
  • The engineered adeno-associated viruses (AAVs) reached up to 71 percent of neurons and 92 percent of astrocytes in different brain regions.


Gene therapy has the potential to treat severe genetic brain disorders that currently lack cures and effective treatment options. However, existing adeno-associated viruses (AAVs), commonly used for gene therapy, struggle to efficiently cross the blood-brain barrier.

The Breakthrough

Researchers at the Broad Institute of MIT and Harvard have developed an AAV that targets a human protein expressed in the blood-brain barrier. By binding to the human transferrin receptor, this engineered AAV can reach the brain at much higher levels than the FDA-approved AAV9 used in central nervous system gene therapy.

Importantly, the new AAV also reaches a large fraction of brain cells, including neurons and astrocytes. This opens up exciting possibilities for treating brain diseases such as Gaucher's disease, Lewy body dementia, and Parkinson's disease.


The breakthrough in gene therapy delivery represents a significant step toward more effective treatments for brain disorders. By harnessing the power of engineered AAVs, scientists are paving the way for a future where safer and targeted therapies can address previously untreatable conditions.

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