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The AAV Question Gene4Neuro Series Ask a group of people working in gene therapy for neurological disease which vector they would bet on, and a substantial fraction will still say adeno-associated virus. There are good reasons for this. AAV's track record is comparatively strong: decades of clinical safety data, established manufacturing infrastructure, tissue tropism with some serotypes, such as AAV9, with preference for the nervous system, and a regulatory pathway that

Gene therapy for the brain has arrived. What happens next is a conversation the whole field needs to have. Broadreach Global · Gene4Neuro Series · Opening post · A decade ago, if you told a neurologist that a single intravenous infusion could halt the progression of a fatal infant disease — reprogram a faulty gene, and let a child walk who would otherwise never have stood — they might have called it science fiction. It isn't. It happened. And for those of us working at the in