In vivo CAR-T therapy marks a paradigm shift by generating CAR-T cells directly inside patients through advanced gene delivery. This article presents a comprehensive and up-to-date synthesis of the field, integrating delivery platform engineering, clinical progress, and translational challenges. By comparing viral and non-viral strategies and summarizing global trials in cancer and autoimmune diseases, it provides a roadmap for clinical development.

An Australian-led international research collaboration has delivered a promising breakthrough in the quest to better understand and treat childhood dementia.

Recently published in one of the world’s most preeminent scientific journals, Nature Communications, the study uncovered a fundamental mechanism underlying Sanfilippo syndrome, a common form of childhood dementia, revealing how hyperactive and dysregulated synaptic circuits emerge in the brain tissue of children impacted by this devastating disease.

Researchers at Umeå University have contributed new insights into how cancer cells protect themselves from cell death. The study provides a deeper understanding of how key proteins interact within the cell and could, in the long term, support the development of new cancer therapies.

Neuroblastoma kills more children under one year of age than any other extracranial solid tumor, and high-risk cases have resisted meaningful improvement in survival for decades. A team at the Hebrew University of Jerusalem has now identified a molecular accomplice: neuronal nitric oxide synthase, or nNOS, which feeds the mTOR growth-signaling pathway through nitrosative stress. Blocking nNOS, either pharmacologically with the compound BA-101 or genetically with siRNA, silenced mTOR signaling and crippled malignant behavior in human neuroblastoma cells. In a xenograft mouse model, BA-101 shrank tumors dramatically (p < 0.001). The nNOS–mTOR axis emerges as a new and targetable vulnerability. NeuroNOS Ltd., which partly funded this work, has obtained a license for the patent applications of the BA-101 molecule filed by Yissum (The Hebrew University Technology Transfer Company). The authors, in collaboration with NeuroNOS, have also demonstrated the therapeutic efficacy of BA-101 in glioblastoma.

More than three-quarters of all cases of liver cancer worldwide are associated with chronic viral hepatitis but scientists have been limited in their ability to model how these viruses lead to cancer. In the new study, a Rockefeller team showed that mice infected with an engineered version of a rat virus develop liver inflammation, scarring, and ultimately cancer similar to that seen in humans with viral hepatitis-associated liver cancer. The new mouse model can be used to study how liver virus infection leads to cancer as well as to test treatments.