Gene-Delivering Viruses Reach the Brain in Step Toward Gene Therapy for Neurological Diseases
Researchers have developed a family of adeno-associated viral vectors (AAVs) that are able to cross the blood-brain barrier to deliver gene therapies directly to the brain.
Gene therapies can treat, even potentially cure, certain genetic diseases, but it is challenging to deliver the treatments to the parts of the body where they are needed.
Researchers have engineered viruses called adeno-associated viruses (AAVs) to deliver cargo — such as a functioning copy of a gene — to specific cells and organs, but they don’t always get to their desired destination.
Researchers at the Broad Institute of MIT and Harvard have now developed a family of AAVs that is able to reach a particularly challenging target tissue — the brain. The team shows, in a study published in Med, that their AAVs are more than three times better at delivering their cargo into the primate brain than the current leading AAV delivery vehicle, AAV9.
The new AAVs can cross the blood-brain barrier, which keeps many drugs from getting into the brain. They also accumulate much less in the liver than AAV9, potentially reducing the risk of liver side effects that have been seen in other AAV9-based gene therapies. This family of AAVs, called the PAL family, could be a safer and more efficient way to deliver gene therapies to the brain.
The AAVs were designed in the lab of Pardis Sabeti, who is an institute member at the Broad, a professor at Harvard University and the Harvard T.H. Chan School of Public Health, and a Howard Hughes Medical Institute investigator.
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