Article
Molecular basis of engineered meganuclease targeting of the endogenous human RAG1 locus.
Nucleic acids research - 1 Jan 2011
Muñoz Inés G, Prieto Jesús, Subramanian Sunita, Coloma Javier, Redondo Pilar, Villate Maider, Merino Nekane, Marenchino Marco, D'Abramo Marco, Gervasio Francesco L, Grizot Sylvestre, Daboussi Fayza, Smith Julianne, Chion-Sotinel Isabelle, Pâques Frédéric, Duchateau Philippe, Alibés Andreu, Stricher François, Serrano Luis, Blanco Francisco J, Montoya Guillermo
Abstract excerpt
Homing endonucleases recognize long target DNA sequences generating an accurate double-strand break that promotes gene targeting through homologous recombination. We have modified the homodimeric I-CreI endonuclease through protein engineering to target a specific DNA sequence within the human RAG1 gene. Mutations in RAG1 produce severe combined immunodeficiency (SCID), a monogenic disease leading to defective...
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