Homology independent targeted integration for gene editing
AssigneeFondazione Telethon Ets
InventorsAlberto Auricchio, Fabio Dell'Aquila, Federica Esposito, Rita Ferla, Manel Llado Santaeularia
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AssigneeFondazione Telethon Ets
InventorsAlberto Auricchio, Fabio Dell'Aquila, Federica Esposito, Rita Ferla, Manel Llado Santaeularia
Estimating the term…
The present invention relates to a method, preferably a homology independent targeted integration (HITI), of integrating an exogenous DNA sequence into a genome of a cell comprising contacting the cell with a donor nucleic acid comprising said exogenous DNA sequence, optionally one or more albumin exons, wherein said donor nucleic acid is flanked at 5′ and 3′ by inverted targeting sequences; a complementary strand oligonucleotide homologous to the targeting sequence and a nuclease that recognizes the targeting sequence, wherein said targeting sequence is located at the 3′ end of the albumin gene in a region selected from intron 12, intron 13 and intron 14 of said albumin gene. The invention also relates to systems, vectors and pharmaceutical compositions comprising said donor nucleic acid and/or complementary strand oligonucleotide homologous to the targeting sequence and/or nuclease and to medical uses thereof.
A method of integrating an exogenous DNA sequence into a genome of a cell comprising contacting the cell with:
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A system comprising:
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Dependencies are inferred from the claim text.