Only one individual in history has been cured of HIV. This person is known as “the Berlin patient,” named for the location where the renowned HIV-ridding procedure took place. Scientists at the Oregon National Primate Research Center (ONPRC) at OHSU are working to understand how a specific mutation in a gene may block HIV infection in the host. Using CRISPR technology, researchers are creating the same mutation and bone marrow transplantation performed on that patient to study how it might play a role in HIV infectivity.
Jon Hennebold, professor and chief, Division of Reproductive & Developmental Sciences, at ONPRC, said that Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR) genome-editing technology is responsible for these new insights. “You can’t fully study HIV in rodent models because it’s a primate-tropic virus,” he said. CRISPR is essentially a programmable molecular scissors that scientists can manipulate to edit the genes within organisms. CRISPR/Cas9 seeks and targets specific genes in organisms that are linked to diseases by utilizing a single strand of RNA as a guide to target specific genes for editing.
“This technique exploded in the scientific community about five years ago, so it’s relatively new,” Hennebold said. “Basically, CRISPR works by cutting the DNA in the target gene of interest, which in turn results in the creation of a mutation at that site when the cell repairs the gap in the gene,” Hennebold said. “It doesn’t do it randomly. It goes to a gene of interest and will cut the DNA at that point.” Further, “It was a huge advance from the standpoint of being able to modify genomes, so it could be used to modify many different organisms’ genomes. Previously, you were only able to do that in a few models. You could never apply the previous approaches to organisms other than mice.”
In addition to work with HIV, CRISPR is currently being used to help researchers understand conditions such as blindness, autism, and neurodegenerative diseases that are too complex to be studied in a rodent research model.
