In this study, researchers utilized an AAVrh10 vector to deliver a computationally-designed, thermostable form of GCase (dGCase) directly into the left lateral ventricle of Gba−/−;Gbatg mice, a model for type 3 GD. The results demonstrated significant improvements in the treated mice, including increased body weight, extended lifespan, and enhanced motor function compared to mice receiving the wild-type GCase (wtGCase) via the same vector.
Biochemical analyses revealed a reduction in GlcCer levels and an increase in GCase activity in the brain’s right hemisphere of dGCase-treated mice. Additionally, these mice exhibited reduced neuroinflammation and lower expression of genes typically elevated in neurological forms of GD. These findings suggest that modified enzymes, when delivered using an optimized AAV vector, may offer a more effective therapeutic approach for lysosomal storage diseases and potentially other monogenetic disorders.
This study underscores the potential of AAV vector construction and protein engineering in developing more effective treatments for genetic diseases, particularly those with neurological involvement.
https://www.nature.com/articles/s41434-024-00476-8
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