Scientists at Synlogic Inc. have engineered bacteria to produce phenylalanine hydroxylase (PAH), the gene that is missing or dysfunctional in individuals with phenylketonuria (PKU). Currently in a phase I trial in healthy volunteers and adult phenylketonurics, the bacteria sit at an intersection between synthetic biology and gene correction.

Ovarian cancers, as well as several other difficult-to-treat cancer types, are characterized by complex copy number alterations – that is, they have copy number alterations, but those alterations differ from tumor to tumor. Researchers from the British Cambridge University and Imperial College London have developed a method to classify such tumors by the underlying genomic housekeeping problem that led to their formation. 

Antibody-dependent enhancement (ADE) has long been a thorn in the side of dengue vaccine developers. In the Aug. 14, 2018, issue of Cell Reports, a team from the University of Texas Medical Branch showed that ADE occurred with human antibodies to the Ebola virus as well, but it could be prevented by engineering antibodies to prevent their interaction with immune cells.

It's an unexpected form of optimization for sure. But researchers at the University of Virginia have combined aspects of two antibodies that have failed in clinical trials to make a bispecific antibody that was able to shrink ovarian tumors in vivo.

Researchers at Memorial Sloan-Kettering Cancer Center (MSKCC) have reported both preclinical data and a case study of a patient treated under a compassionate use protocol indicating that the tropomyosin receptor kinase (TRK) fusion inhibitor larotrectinib (LOXO-101, Loxo Oncology Inc.) is effective in hematological malignancies as well as in solid tumors, where it is currently in late-stage clinical trials. 

Scientists at the Cleveland Clinic Foundation have developed small-molecule inhibitors that inhibited the bacterial enzyme chain responsible for processing choline into trimethylamine-N-oxide (TMAO).

Stanford University scientists have shown that homeostatic plasticity was disrupted in fragile X syndrome (FXS) through effects on retinoic acid (RA) signaling. The fragile X mental retardation 1 (FMR1) gene that is mutated in FXS codes for an RNA binding protein with multiple interaction partners.

The gene STK11 encodes the tumor suppressor liver kinase B1 (LKB1), and STK11 mutations promote Peutz–Jeghers syndrome (PJS), a cancer predisposition syndrome. Individuals with PJS are prone to developing gastrointestinal polyps, which can lead to gastrointestinal (GI) tumors. Researchers from the Van Andel Research Institute and the Canadian McGill University have shown that STK11 mutations led to increased inflammation, and that targeting the inflammation could improve outcomes in mouse models of PJS.