A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof
AssigneesCellectis, Precision Genome Engineering, Inc.
InventorsJordan JARJOUR, Alexander ASTRAKHAN
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Abstract
Disclosed herein are compositions for inactivating the human CCR5 gene comprising engineered LAGLIDADG homing endonucleases (LHEs) and their derivatives, particularly derived from members of the \-OnuI subfamily of LHE. Polynucleotides encoding such endonucleases, vectors comprising said polynucleotides, cells comprising or having been treated with such endonucleases, and therapeutic compositions deriving therefrom are also provided.
Claims25
An I-OnuI or I-OnuI homologue, in which mutations have been introduced to obtain a variant that cleaves a target nucleic acid sequence within the C-C chemokine receptor type 5 gene (CCR5).
Dependencies are inferred from the claim text.