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Sending Genes into the Brain

Sending Genes into the Brain

The brain has long presented a special challenge to drug developers: tightly enclosed by the blood brain barrier, it remains locked to many therapies delivered orally or intravenously.

However, thanks to more-precise methods of targeting the brain, advances in brain imaging, and the growing popularity of implanted stimulators for treating neurological diseases, the brain is no longer off limits. This is highlighted by a number of new clinical trials involving Parkinson’s patients, in which a therapeutic gene or another treatment is delivered directly to a specific part of the brain.

“My belief is that we’re entering into an era where instrumentation in the brain will become routine, not just for Parkinson’s, but for myriad central nervous system disorders,” says Howard Federoff, a neurologist and executive dean of the School of Medicine at Georgetown University, in Washington, DC. “I anticipate that delivery technologies will drive the development of new therapeutics and the repurposing of existing treatments, where they could be delivered directly to the part of the brain where it’s needed at the appropriate dose.”


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