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Author

Harrison Higgs

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Open access Oct 2026

A minimally modified AAV9 variant (AAV9fyhr) for enhanced CNS gene delivery by intra-CSF administration

Adeno-associated virus (AAV) mediated gene therapies continue to show promise for the treatment of genetic diseases. However, delivery and safety still represent significant prohibitive barriers restricting translation of these delivery modalities to humans. We describe a novel AAV9 variant, “AAV9fyhr” or “FYHR”, ident...

Irvin T. Garza, V. Ramesh, Yu-Hui Hu et al. · 0 citations
Open access Sep 2026

Expanding clinical and variant spectrum of CBX1 -related syndrome: Report of three novel cases

In vitro cellular assays revealed that CBX1 p.(Cys60Gly) variant showed an intermediate cellular phenotype between the wild type and the CBX1 p.(Leu40Pro) and p.(Cys60Arg) variants, suggesting a possible genotype-phenotype correlation.

Graciela Carrillo, Shuhei Fujino, Harrison Higgs et al. · 0 citations

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