Pompe disease is caused by a deficiency in the lysosomal enzyme acid α-glucosidase (GAA) that leads to accumulation of glycogen in the lysosomes, mainly seen in skeletal and cardiac muscles. Researchers from Duke University have developed a new murine model of Pompe disease, which recapitulates human infantile-onset disease. This model harbors the c.1826dupA mutation in the murine Gaa gene, which resembles the human GAA c.1826dupA (p.Y609*) mutation seen in infantile-onset Pompe disease.
Traditionally developed antibiotics generally act inhibiting essential bacterial enzymes. However, new strategies are urgently needed to discover novel antibiotics against bacterial infections, such as Lyme disease caused by Borrelia burgdorferi. The chaperone high-temperature protein G (HtpG) is a nonessential bacterial protein containing a desirable druggable domain.
Adaptative immune response mediated by NKG2D receptor and its ligand NKG2DL could be the clue for CD8-expressing “killer” T cells to kill tumors lacking the major histocompatibility complex (MHC) class I, according to a group of researchers at Duke University.
A different class of antibiotics could ease the increasing resistance triggered by some gram-negative bacteria. LpxC inhibitors are not new, but all attempts to develop them have failed due to cardiovascular toxicity or ineffectiveness. A modification of the structure of these compounds may have solved the problem. Duke University scientists demonstrated the preclinical safety and efficacy of an LpxC inhibitor candidate against a wide selection of these pathogens.
Researchers from Duke University presented the discovery and preclinical evaluation of [211At]YF-2, a next-generation prostate-specific membrane antigen (PSMA)-targeted radiopharmaceutical.
Research led by Duke University and the German Center for Diabetes Research shows the glucose-dependent insulinotropic polypeptide (GIP) receptor plays an essential role in the action of the type 2 diabetes drug Mounjaro (tirzepatide; Eli Lilly and Co.) in human pancreatic islets.
Currently, most patients with urinary tract infections (UTIs) require long-term antibiotic treatment, which poses risks of resistance development and alters the patients' microbiota. Therefore, vaccines that raise safe, effective and long-lasting immune responses across populations for UTI prevention constitute a critical medical need. However, developing such vaccination strategies remains challenging because the responses need to be specific to a broad range of UTI-causing bacteria and include both blood and mucosal responses in the urogenital tract.
A new vaccine that uses the native-like HIV-1 envelope (Env) trimer CH505 and a Toll-like receptor (TLR) 7/8 agonist adjuvant, successfully evaluated in macaques, generated potent polyclonal neutralizing antibodies (nAbs) and a high protection against the infection of the homologous simian-human immunodeficiency virus (SHIV).
Tune Therapeutics Inc. launched with $40 million in initial financing and plans to pursue both rare and complex disease indications with a series of epigenetic editors that employ CRISPR-Cas9 DNA recognition to modulate gene expression in a targeted fashion, without introducing potentially problematic DNA strand breaks or changes to the genetic code.