Correction of exon skipping in monocyte-derived cells for improved immune response
OwnerD. Christopher Schellinginventor
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Abstract
The present disclosure relates generally to compositions and methods for inhibiting exon 3 skipping in the 6-pyruvoyltetrahydropterin synthase (PTS) gene in monocytes, monocyte-derived cells such as macrophages and dendritic cells of the precursor cells thereof. The inhibition can be achieved with genome editing of the genomic sequence to remove certain splicing factor recognition sites or inhibiting the expression or activity of the splicing factors that contribute to the cell-specific exon skipping. Monocytes and monocyte-derived cells that have reduced exon skipping in the PTS gene can generate more potent immune response and thus are useful in preventing or treating diseases such as infectious diseases and cancer.
Claims20
A method for inhibiting or correcting the skipping of exon 3 in the 6-pyruvoyltetrahydropterin synthase (PTS) mRNA or promoting recognition of the exon 3 in a cell, comprising editing the PTS genomic sequence of pre-mRNA, or contacting the cell with an agent that inhibits exon skipping or promotes exon recognition or inclusion.
A method for modulating the immune system in a mammalian subject in need thereof, comprising editing the 6-pyruvoyltetrahydropterin synthase (PTS) genomic sequence or pre-mRNA in a monocyte, a macrophage, a dendritic cell or a precursor cell thereof in the mammalian subject, wherein the editing inhibits skipping of exon 3 in the PTS mRNA during splicing.
A method for modulating the immune system in a mammalian subject in need thereof, comprising administering to the subject an agent that inhibits exon 3 skipping or promotes exon 3 recognition in the 6-pyruvoyltetrahydropterin synthase (PTS) pre-mRNA in a monocyte, a macrophage, a dendritic cell or a precursor cell thereof in the subject, or administering a nonsense suppression agent to a tissue in the subject that produces or circulates monocytes, macrophages, dendritic cells or precursor cells thereof
Dependencies are inferred from the claim text.