University of Rochester Medicine researchers have developed a gene therapy platform designed to deliver therapeutic genes broadly through the brain while targeting glial cells.
The approach uses the brain's glymphatic transport system and engineered viral vectors, a combination researchers say could make future treatments for neurological diseases safer and more effective.
The study was published in Nature Biotechnology and led by Steve Goldman, co-director of the University of Rochester Medicine Center for Translational Neuromedicine.
Goldman said gene delivery to the brain has long faced two major obstacles: getting therapies into the brain efficiently and ensuring vectors deliver those therapies to the right cells once inside.
The platform pairs engineered adeno-associated viruses with a delivery strategy that uses the brain's natural fluid transport pathways. Researchers said the approach allowed therapeutic genes to spread broadly through the brain while preferentially targeting human glial cells and limiting exposure to other tissues.
Glial cells are support cells in the nervous system that help maintain brain function, produce myelin and regulate neuronal health. Goldman said many neurological disorders involve glial dysfunction, creating a need for tools that can safely deliver therapies to those cells throughout the brain.
To develop the platform, researchers engineered a library of modified AAV5 viral vectors and screened them in mice whose brains had been transplanted with human glial progenitor cells.
The resulting vectors preferentially targeted human glial progenitor cells and their descendants, including astrocytes and oligodendrocytes, while showing limited infection of peripheral tissues.
Researchers said the approach may eventually help treat disorders tied to glial cells and white matter, including multiple sclerosis, Huntington's disease and rare childhood neurological disorders.
Goldman's team is also exploring the use of artificial intelligence to design viral capsids with specific targeting characteristics, potentially accelerating the development of future gene therapies.
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