ANOKIWIN, Japan — Researchers at the AnokiiWin training institute say they have developed a bloodhound gene therapy that can change the brain in mice.
The institute said the gene therapy can be used in humans as soon as 2019, and it has been applied to mice.
Gene therapy, or gene-editing, is a form of genetic engineering that involves modifying a gene to create new ones.
The technology can also be used to help humans.
Anokii has been working on gene therapy for two decades, and its research is in the realm of advanced medicine.
But its first gene therapy, an engineered mouse gene called BHLH, could open up a new avenue for gene therapy.
Scientists at the institute say their new gene therapy was developed using a technique called a chimeric antigen receptor (CAR), which is a type of immune cell that can recognize and bind specific antibodies.
CARs are used in cancer therapies.
The institute says the CARs that are targeted in the gene-mediated therapy were engineered to make them more easily translatable to a mouse, which was then bred to be genetically identical to human.
This allows for a more effective delivery method for the gene.
The new gene is also made from human embryonic stem cells, or ESCs.
“We believe that our CAR-based gene therapy will have a very large therapeutic impact in humans,” Anoki Win founder Yoshiro Kawakami told The Associated Press by phone from Japan.
In addition to changing the gene expression in mice, the gene treatment can also help patients with Huntington’s disease, diabetes and arthritis.
The researchers say they’ve used the gene to treat Alzheimer’s disease in mice and a number of other disorders, and that it has shown promise in humans.
The institute says its gene therapy could eventually be used for other disorders including cancer.
Kawakami says he and his team have already received a number the first batch of the treatment’s cells, and are preparing to test its safety and efficacy in humans, where they hope to begin human trials in 2021.
Copyright Associated Press / NBC News Bay Area