CRISPR-MEDIATED HUMAN GENOME EDITING WITH VECTORS
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United States of America
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Issued Date -
Apr 3, 2025
app pub date -
Jan 25, 2023
filing date -
Jan 25, 2023
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Abstract
A Cas based system for gene therapy is provided. For example, a method is provided to prevent, inhibit or treat one or more symptoms so GM1-gangliosidosis in a mammal, comprising: administering to the mammal an effective amount of i) Cas or an isolated nucleic encoding Cas, and ii) isolated nucleic acid for one or more gRNAs comprising a targeting sequence for a mammalian genomic target and nucleic acid comprising a coding sequence for beta-galactosidase flanked by homology arms that bind to the mammalian genomic target is disclosed.
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