Can gene therapy be used to knock out a gene?

Effects of genome interventions depend on the methods used. ... However, it has been shown that consequences can differ, after a gene is either knocked, out or only blocked. ... Before we establish such therapies, we have to fully understand the consequences the loss or blockade of a gene might have. read more

The invention of Crispr/Cas9 makes it easier to knock out a gene in gene therapies. In Nov 2016, the first clinical trial of Crispr/Cas9 gene therapy was launched in Sichuan, China. You Lu, the leading scientist, also a physician, used Crispr/Cas9 to knockout PD-1 gene in T cells to increase immune response. read more

A gene knockout (abbreviation: KO) is a genetic technique in which one of an organism's genes is made inoperative ("knocked out" of the organism). However, KO can also refer to the gene that is knocked out or the organism that carries the gene knockout. read more

Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein. read more