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 and in vivo in a mouse model that produces some functional protein, correcting the altered gene expression and improving neuronal functions.

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.

CAR T cells have been groundbreaking for the treatment of B-cell cancers. But 8 years after Kymriah (tisagenlecleucel, Novartis AG) became the first CAR T-cell therapy to be approved, there are no CAR Ts approved for solid tumors.

The massive cuts to science, global health, and HIV programs that unfolded in 2025 triggered a crisis with worldwide repercussions. The dissolution of USAID, the shutdown of PEPFAR, and the suspension of thousands of NIH research projects led to an immediate collapse of essential services, from HIV prevention to access to treatment. At the 33rd Conference on Retroviruses and Opportunistic Infections (CROI) held Feb. 22-25, 2026, in Denver, scientists, activists, and health professionals presented data illustrating the scale of the damage and warned of a historic setback in the global HIV response.

The effects of aging pose an additional challenge for people with HIV due to the neurological and psychological consequences that persist despite antiretroviral therapy. At the Conference on Retroviruses and Opportunistic Infections (CROI) held Feb. 22-25, 2026, in Denver, the scientific community examined how the virus affects the brain, how the reservoir is established in the CNS, and which genetic, immunological or treatment-related factors influence cognitive health.

Antiretroviral therapies against HIV have been in use for more than 30 years and have enabled people living with HIV to maintain undetectable viral levels. Many of them are aging in good health. However, others present symptoms of cognitive decline. HIV can reach the brain and establish a reservoir there. Yet, it is still unknown what this reservoir is like, which cells are affected, and which comorbidities are typical of aging or are associated with the virus.

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 malfunction of lymphatic drainage that occurs in lymphedema leads to excessive cholesterol accumulation in the affected skin and lymphatic vessels, causing inflammation and fibrosis. However, surgery and the chemical reduction of accumulated cholesterol with cyclodextrin reduce inflammation and regenerate lymphatic vessels. Cholesterol is a potential therapeutic target for treating lymphedema, according to this study published on Feb. 11, 2026, in Nature by Veronique Angeli and her colleagues.

Hepatocellular carcinoma (HCC) is the most common form of primary liver cancer, representing approximately 75-85% of all cases. Often considered preventable, primary liver cancer ranks as the sixth most frequently diagnosed cancer and the third leading cause of cancer deaths worldwide. Through a multi-institutional effort, researchers have identified activated ATF6α as a driver of HCC that suppresses immune defenses, predicts response to immune checkpoint therapy, and represents a potential target for intervention.

The neural and neuroimmune mechanisms behind myocardial infarction-triggered cardiac events, immune responses and activation of the nervous system remain largely unexplored. The heart and the brain talk to each other in what is known as cardioception. This communication between the two organs is orchestrated through neurons of the vagus nerve or the dorsal root ganglia, among others. Researchers from the University of California, San Diego have now shown that the dynamics of these interactions may play a crucial role in modulating inflammation, repair and cardiac functioning.