Medicine

An experimental gene therapy enabled surviving retinal neurons to respond to light in people with advanced retinitis pigmentosa. Seven of ten participants showed improved light sensitivity, while some gained limited object-detection abilities using specialized goggles.

A single infusion of an experimental CRISPR gene-editing therapy reduced hereditary angioedema attacks by 87% compared with placebo. Phase 3 results suggest that disabling a gene in liver cells could provide lasting disease control without continuous preventive medication.

Targeted lipid nanoparticles delivered mRNA directly to NK cells in mice, temporarily creating CAR NK cells inside the body. The platform attacked tumors and autoreactive T cells without ex vivo cell manufacturing.

A bacterial metabolite produced in the gut altered immune-cell behavior and experimental stroke severity in mice. Human data pointed in the same direction, but remained observational and did not establish the same causal pathway.

Twenty people with early-stage Huntington's disease followed a 6–8-hour daily eating window for 12 weeks. Weight remained stable, while plasma neurofilament light, a marker of neuroaxonal damage, fell by an average of 13%.

Psilocybin given before chemotherapy protected peripheral nerves in mice. The effect was linked to preservation of mitochondrial transport along sensory nerve fibers.

A pill does not search for the painful organ. After absorption, drug molecules circulate through the body, enter accessible tissues, interact with molecular targets, and are eventually metabolized and eliminated.

Why does acute pain sometimes persist after an injury? In mice, researchers traced a pathway from altered oligodendrocyte metabolism and myelin disruption to Aβ42 production—and were able to block the transition to persistent pain.

Doctors injected mitochondria isolated from a 26-year-old patient’s own muscle into both eyes. Vision did not measurably recover, but objective pupillary responses to light temporarily returned after treatment.

A computer-vision system analysed live endoscopic video during pituitary surgery. In the first six-patient series it ran successfully in four cases, with no AI-attributable complications, but clinical benefit has not yet been established.

Chemically modified suppressor tRNA helped cells read through premature stop codons and restore full-length CFTR in preclinical cystic fibrosis models without editing genomic DNA.

A new study suggests that after brief intestinal inflammation, the extracellular matrix can stay altered for more than a year and keep reshaping how stem cells repair the gut lining. That may help explain how short-term damage can turn into chronic disease.

Longitudinal MRI data show that cognitively healthy people who later became amyloid-positive had a relatively thicker cortex and slower age-related thinning, with differences detectable years before PET positivity.

Oral vitamin D has produced little consistent benefit in chronic lung disease. A new Perspective argues that direct delivery to the airways may be worth testing, based on mechanistic and preclinical evidence.

The FDA has approved mFlusiva, the first U.S. seasonal mRNA flu vaccine for adults 50 and older. In a large phase 3 trial, it prevented more confirmed flu cases than a standard-dose vaccine, but the headline 26.6% figure is a relative difference.

One of four participants in the first HG302 trial for Duchenne muscular dystrophy died after receiving the high dose. Available evidence points to a severe immune response to systemic AAV delivery rather than demonstrated CRISPR off-target editing.

A 61-year-old woman developed cognitive decline, hallucinations, seizures and severe loss of independence over five years. The underlying cause was impaired vitamin B12 absorption, and treatment restored much of her cognitive and everyday functioning.

A recent Bourbon virus infection was confirmed in a Suffolk County patient through a sharp rise in neutralizing antibodies. The study reveals how rare tick-borne viruses can remain hidden behind more familiar diagnoses.

Bernhard Zondek helped turn early pregnancy from a clinical suspicion into a laboratory finding. His mouse bioassay detected hormonal activity in urine and established the principle behind modern pregnancy tests.

A woman taking semaglutide developed a dense gastric bezoar. Doctors used an unusual treatment—about 1.5 liters of diet cola per day—and the mass was no longer present on repeat endoscopy.

An archival photograph shows a nurse sharing news of the Salk vaccine trial with a patient who had already been disabled by polio. The story explains what the 1.8-million-child trial proved—and what it could not yet answer.

Carbon dioxide is normally treated as a waste gas that the body needs to expel. Yet brief, carefully controlled increases in CO₂ produced a different effect in the brain: blood vessels…

mRNA vaccines have a confirmed rare adverse effect: inflammation of the heart muscle or its surrounding membrane, seen most often in younger males after a second dose. One of the most…