Gene Therapy Approach Shows Promise for Hydrocephalus

Researchers develop AAV-based method to target cerebrospinal fluid production, revealing new insights into postnatal neurogenesis

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Researchers developed an AAV5-CMV-DTR platform to target choroid plexus epithelial cells and reduce cerebrospinal fluid production in mice.
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In adult mice with kaolin-induced hydrocephalus, treatment within 6 to 10 hours reduced brain edema and corpus callosum MRI abnormalities.
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Treatment did not substantially reduce ventricular enlargement or cause ventricular collapse in the adult mouse model.
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In neonatal mice, choroid plexus ablation reduced neuroblast populations in the subventricular and hippocampal subgranular zones.
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Neonatal treatment was associated with smaller hippocampal and overall brain volumes and modestly reduced exploratory locomotor activity.

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