The human pancreas often contains lesions that are common in the population and that, although they can become malignant, usually do not. To understand when this occurs and how these pancreatic cancer precursor lesions, known as pancreatic intraepithelial neoplasias (PanIN), evolve, scientists at the University of Michigan analyzed their gene expression patterns and their cellular microenvironment.

Recent findings are reshaping current understanding of the post-infection landscape of SARS-CoV-2. Although previous studies had already suggested that autoimmunity might underlie the persistent neurological symptoms seen in long COVID, researchers at Yale University and Mount Sinai now reinforce this hypothesis. SARS-CoV-2 infection appears to trigger an autoimmune mechanism that drives chronic pain, fatigue and cognitive impairment in some patients.

WAVES, an algorithm designed to extract menstrual-cycle metrics from physiological signals such as basal body temperature, which oscillates with sex hormones, shows how different parameters change with age and helps determine whether each person maintains a stable individual pattern or personal footprint. A study based on data from 5,674 cycles from 753 women demonstrates through this tool that age is associated with higher temperatures, shorter cycles, and greater irregularity. In addition, several metrics show within-person stability, suggesting they could serve as personalized health markers.

A new strategy aims to improve gene therapy for Pompe disease by optimizing both the genetic component that restores the function of a deficient lysosomal enzyme and the vector that delivers it to the target tissue while avoiding the liver. The findings suggest that combining an optimized transgene with a targeted capsid could significantly enhance the effectiveness of gene therapy for Pompe disease.

Directed evolution has become a central pillar in gene therapy. This engineering strategy enables the generation of more efficient variants of genetic editors and delivery vectors. Molecular diversification methods are increasingly sophisticated and are now accelerated by machine learning and AI tools, as showcased at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) held in Boston this week.

Gene therapies rely on vectors to reach the target tissue where they act, such as adeno-associated viruses (AAVs) or lipid nanoparticles (LNPs), among other delivery strategies. Each combination is optimized for a specific cell type and indication, aiming to overcome challenges such as efficacy, specificity and toxicity. On May 13, 2026, two sessions included in the scientific symposia of the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), being held in Boston this week, addressed AAV-related toxicities, which have led to fatal cases in clinical trials and remain an area for improvement in approved therapies.

Circular RNA (circRNA) is not a new concept, but it is a novel strategy in the field of gene and cell therapy. While mRNA vaccines have revolutionized medicine, this RNA fragment without free ends surpasses their performance in both efficacy and durability, bringing it to the attention of several pioneering companies. The latest advances in circRNA presented at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) clearly surpass the performance achieved with linear mRNA.

A designed chimeric virus induced broadly neutralizing antibodies against the macaque equivalent of HIV. The strategy works in two steps: first it uses an envelope protein with a mutation that reduces the glycan shield that makes it invisible to the immune system, and then it exposes the part of the protein most likely to generate these antibodies capable of blocking many variants of the virus. The macaques developed potent and diverse antibodies with this approach, which pave the way for the development of an HIV-1 vaccine.

A new mRNA and lipid nanoparticle (mRNA-LNP) platform could selectively reprogram in vivo cytotoxic effector T cells (Teff), the cells responsible for eliminating infected or tumor cells. To achieve this, scientists at the University of Pennsylvania conjugated LNPs with fractalkine, a molecule that binds to the CX3CR1 receptor, which is a marker of Teff cells. Using this strategy, the researchers delivered an mRNA encoding new proteins such as IL‑2 or human CD62 L‑selectin, opening the door to temporarily reprogramming these cells within the body, both in the blood and in lymphoid tissue, where they reside and become activated.

A new mRNA and lipid nanoparticle (mRNA-LNP) platform could selectively reprogram in vivo cytotoxic effector T cells (Teff), the cells responsible for eliminating infected or tumor cells. To achieve this, scientists at the University of Pennsylvania conjugated LNPs with fractalkine, a molecule that binds to the CX3CR1 receptor, which is a marker of Teff cells. Using this strategy, the researchers delivered an mRNA encoding new proteins such as IL‑2 or human CD62 L‑selectin, opening the door to temporarily reprogramming these cells within the body, both in the blood and in lymphoid tissue, where they reside and become activated.