Similarities among three pediatric brain tumors that arise in different structures of the CNS – pineoblastoma, retinoblastoma and Group 3 medulloblastoma – have been linked to their shared origin during pineal gland development. Scientists at St. Jude Children’s Research Hospital have identified the molecular signatures that drive these tumors from pinealocyte progenitor cells that conserve a common differentiation program, providing a shared therapeutic target for these three cancer types.

If one could sweep the brain clean and send the toxic substances that drive neurodegeneration to the recycling bin, perhaps one could treat Alzheimer’s disease (AD). Researchers at the Chinese Academy of Sciences propose a new therapeutic strategy that uses synthetic peptides that bind to amyloid-β (Aβ) and direct it toward lysosomes. In addition, researchers at the Washington University School of Medicine in St. Louis have genetically modified astrocytes in vivo to express chimeric antigen receptors (CARs) that recognize and phagocytose Aβ plaques.

A therapeutic strategy based on alternative splicing of the MECP2 gene could restore protein levels in Rett syndrome, a neurological disorder caused by mutations in that gene. Scientists at Baylor College of Medicine have successfully tested this approach both in vitro in neurons from Rett patients that produce some functional protein, correcting the altered gene expression and improving neuronal functions, and in vivo in mice.

Computational pathology, which assesses molecular-level features of diseases directly from tissue images (rather than testing the tissue via methods such as staining or sequencing) is making rapid strides.

A project that started as a bioreactor to assist astronauts in deep space to keep medications safe in a microgravity environment could help pharma companies model how drugs behave in the human body. Omnigeniq unveiled at the J.P. Morgan Healthcare conference the first computer model of a human protein as it exists in the body, confirming that native protein topology can be calculated directly from physics.

Parkinson’s disease is a neurodegenerative disorder that affects movement, and tremor is one of its signatures. But it is a much more wide-ranging disorder, and patients experience problems with cognitive and emotional processes as well. SCAN, the somato-cognitive action network identified in 2023, could reshape the definition of PD. Treating this circuit can improve outcomes.

The variety of organoids that can be developed in vitro is enabling major advances. Depending on the type of tissues and the research goals, these small 3D cell-based structures that mimic real tissue offer certain advantages over animal models. Scientists at the University of Padova in Italy have created human neuromuscular organoids to reproduce cancer-induced muscle cachexia, a condition that murine models do not accurately replicate.

The use of DNA scaffolds could mark a turning point in HIV vaccine design. Scientists at Scripps Research and the Massachusetts Institute of Technology (MIT) have created a new vaccine platform based on DNA origami, a material that the immune system does not recognize as a threat, avoiding unwanted responses.

Galux closed a ₩42 billion (US$29 million) series B round Feb. 10, led by Yuanta Investment to bring AI-driven “rational design” to the protein drug development process, already heavily influenced by human engineering.

Parkinson’s disease (PD) is a neurodegenerative disorder that affects movement, and tremor is one of its signatures. But it is a much more wide-ranging disorder, and patients experience problems with cognitive and emotional processes as well. SCAN, the somato-cognitive action network identified in 2023, could reshape the definition of PD. Treating this circuit can improve outcomes.