GeneCopoeia Genome-TALER Human AAVS1 Safe Harbor Knock-in ORF Clones

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The modification of the human genome by the insertion of genes of interest and other genetic elements in unique sites of chromosomes is of great value for cell engineering. The genetically modified cells are valuable for therapeutic research and gene function study, as well as lineage tracking and analysis.

All these applications depend on the reliable and predictable function of the transgene without perturbing any endogenous gene and/or other regulation element. Random integration of the transgene, on the contrary, can present a threat of unpredicted insertion or mutagenesis.

The approach recently developed is to deliver the transgene to a predetermined and safe site in a genome. AAVS1 (also known as PPP1R2C locus) in human chromosome 19 is a well-validated ‘safe harbour’ to host the DNA fragment with expected function. It has an open chromatin structure and is transcription competent. There is no known adverse effect on the cell resulting from the inserted DNA fragment of interest.

Genome-TALER Human AAVS1 Safe Harbor Knock-in ORF Clones from GeneCopoeia are constructed for specifically transferring the ORF of interest from a donor plasmid into the AAVS1 safe harbour site in human chromosome 19 using the Genome-TALER Human AAVS1 Safe Harbor Gene Knock-in Kit. The kit uses TALEN-mediated homologous recombination (HR), which is a natural DNA repair mechanism that occurs in response to DNA double-strand breaks (DSBs). The DSBs here are created by AAVS1-specific TALENs. More than 18,000 sequence-validated human ORFs are available.