Article
mRNA transfection of a novel TAL effector nuclease (TALEN) facilitates efficient knockout of HIV co-receptor CCR5.
Nucleic acids research - 23 Jun 2015
Mock Ulrike, Machowicz Rafał, Hauber Ilona, Horn Stefan, Abramowski Pierre, Berdien Belinda, Hauber Joachim, Fehse Boris
Abstract excerpt
Homozygosity for a natural deletion variant of the HIV-coreceptor molecule CCR5, CCR5Δ32, confers resistance toward HIV infection. Allogeneic stem cell transplantation from a CCR5Δ32-homozygous donor has resulted in the first cure from HIV ('Berlin patient'). Based thereon, genetic disruption of CCR5 using designer nucleases was proposed as a promising HIV gene-therapy approach. Here we introduce a novel...
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