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Targeted Gene Alteration
Targeted gene alteration (TGA) targets and corrects single base-pair mutations in genes by harnessing the DNA repair mechanism in cells. The body is constantly bombarded by chemical and physical insults that lead to mutations in the DNA. Fortunately, each cell in the body is continuously correcting these mutations by a process known as DNA repair. TGA takes advantage of the key mechanism that leads to DNA repair called homologous recombination. OrphageniX takes advantage of this natural repair process by directing the cell to repair point mutation that lead to diseases.

Targeted gene alteration is initiated by delivering specially designed, single stranded oligonucleotides into the nucleus of a cell. Once in the nucleus, the oligonucleotide undergoes a homologous alignment with the complementary sequence in the target gene.

The TGA oligonucleotide is designed to complement perfectly with the target sequence with the exception of a single, mismatched base pair in the center of the complex. The mismatch is a signal for the cell to direct repair enzymes to identify and correct the error. The cell ultimately repairs the target base in the original DNA sequence.

See publications for additional peer reviewed details about Targeted Gene Alteration.