Genetically Modified Primates Offer Hope for Hepatitis B Treatment; CRISPR Activates Suppressed Genes to Treat Genetic Diseases; Guardian Molecule Prevents Liver Cancer

Researchers at Oregon Health & Science University have engineered the first transgenic nonhuman primate model for studying hepatitis B virus (HBV), using a human gene. The study, published in the Proceedings of the National Academy of Sciences, could lead to new treatments for the disease, which affects approximately 250 million people globally, causing nearly 1 million deaths annually. The team inserted the human protein NTCP [N-T-C-P] into the genetic material of rhesus macaques [R-H-E-S-U-S M-A-C-A-Q-U-E-S], making them susceptible to HBV. Lauren Rust, Ph.D. [P-H-D], lead author of the study, stated that liver cells from these models can be infected with HBV in lab tests without additional modifications. Biomedical engineers at Duke University have demonstrated a new approach using CRISPR [C-R-I-S-P-R] to treat genetic diseases like Prader-Willi syndrome [P-R-A-D-E-R W-I-L-L-I S-Y-N-D-R-O-M-E]. By activating a master epigenetic [E-P-I-G-E-N-E-T-I-C] switch, they can turn on suppressed genes from one parent to compensate for defects in the same genes from the other parent. The results were published in Cell Genomics. Charles Gersbach [C-H-A-R-L-E-S G-E-R-S-B-A-C-H], the John W. Strohbehn [J-O-H-N W S-T-R-O-H-B-E-H-N] Distinguished Professor of Biomedical Engineering at Duke, explained that this approach manipulates a master controller of genes already present in the imprinted region. Researchers from the German Cancer Research Center (DKFZ) [D-K-F-Z], the Hector Institute für Translational Brain Research (HITBR) [H-I-T-B-R], and the European Molecular Biology Laboratory (EMBL) [E-M-B-L] have discovered that the PROX1 [P-R-O-X-1] molecule maintains the identity of liver cells. This finding is significant for cancer research, as changes in cellular identity are a fundamental principle of carcinogenesis [C-A-R-C-I-N-O-G-E-N-E-S-I-S]. Moritz Mall [M-O-R-I-T-Z M-A-L-L], a molecular biologist at DKFZ [D-K-F-Z], stated that PROX1 [P-R-O-X-1] can override the influence of strong cancer drivers and suppress tumor formation in mice, even with the presence of cancer mutations in the p53 [P-53] and Myc [M-Y-C] genes. The study was published in Nature Genetics.