Targeted In Vivo Genome Modification

An in vivo method of modifying a genome of a target cell in a mammalian subject is described. The method includes administering an effective amount of a transducer cell to the subject, where the transducer cell includes a regulated viral vector delivery system (RVVDS) for producing and releasing vir...

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Bibliographic Details
Main Authors Maynard, Lucy, Paulk, Nicole K, Mignardi, Marco, May, Andrew P, Tham, Eleonore, Kumar, Gagandeep Renuka, Leenay, Ryan T
Format Patent
LanguageEnglish
Published 05.05.2022
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Summary:An in vivo method of modifying a genome of a target cell in a mammalian subject is described. The method includes administering an effective amount of a transducer cell to the subject, where the transducer cell includes a regulated viral vector delivery system (RVVDS) for producing and releasing viral transduction particles (VTPs) when the transducer cell is exposed to inducing conditions, and each VTP comprises a nucleic acid encoding a genome modification system (GMS) comprising a genome modification protein and one or more elements that regulate expression or activity of the genome modification protein in a mammalian cell.
Bibliography:Application Number: US202017422173