— This is a CRISPR/Cas9 germline mutation in the KCNJ8 gene, equivalent to a human Cantu Syndrome mutation. The mouse is therefore a genetic model of human Cantu Syndrome. This mouse model can be used for pre-clinical testing of targeted therapy for Cantu Syndrome.
Publication: Cardiovascular consequ…
— This mouse model was generated by using CRISPR/Cas9 to mutate the ABCC9 gene. The mouse model described here could be used as a pre-clinical model for testing Cantu Syndrome therapies.
Publication: Cardiovascular consequences of KATP overactivity in Cantu syndrome
— Disease indication – neuroprotection in stroke/ischemia with potential for treating other neurodegenerative diseases
Drug format – neurosteroid
Drug class – drug repurposing (25-hydroxycholesterol)
Research stage and Preliminary data:
In vitro – The inventors demonstrated…
— Lysosomal storage disorders result in severe, multi-system dysfunction and frequently, early death. Currently, a number of lysosomal storage disorders are treated with enzyme replacement therapy (ERT), which requires periodic infusions of the deficient enzyme. However, in a number of instances the…
— Background: Natural Killer (NK) and T cells are the primary immune cells being explored for cancer immuntherapy. NK cells are relatively short-lived, making the therapy less susceptible to some of the side effects currently associated with using T cells for immunotherapy. Unlike T cells, NK cells ar…
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