Chromosomal modification involving the induction of double-stranded dna cleavage and homologous recombination at the cleavage site
AssigneesInstitut Pasteur, Children's Medical Center Corporation
InventorsANDRÉ CHOULIKA, RICHARD C. MULLIGAN
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Abstract
Methods of modifying, repairing, attenuating and inactivating a gene or other chromosomal DNA in a cell are disclosed. Also disclosed are methods of treating or prophylaxis of a genetic disease in an individual in need thereof. Further disclosed are chimeric restriction endonucleases.
Claims19
A method for treating or prophylaxis of a genetic disease in an individual in need thereof comprising inducing in cells of the individual double stranded cleavage at a site of interest using a chimeric restriction endonuclease, said chimeric restriction endonuclease comprising a DNA binding sequence and a DNA cleavage domain, under conditions appropriate for chromosomal DNA homologous to the region surrounding the site of interest to be introduced into the site of interest and repair of the site of interest.
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A method of modifying a specific sequence in chromosomal DNA of a cell comprising inducing in the cell double stranded cleavage at a site of interest in the specific sequence to be modified using a chimeric restriction endonuclease, said chimeric restriction endonuclease comprising a DNA binding sequence and a DNA cleavage domain, under conditions appropriate for chromosomal DNA homologous to the region surrounding the site of interest to be introduced into the site of interest and modification of the specific sequence.
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A chimeric restriction endonuclease comprising a DNA binding sequence and a DNA cleavage domain.
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Dependencies are inferred from the claim text.