gene-therapy

(Vox) – A medical field that almost died is quietly fixing one disease at a time. In a lab room, a toddler, deaf from birth, sits while a tone plays. There’s no reaction. His face does not change. Six weeks … Read More

Intestinal ischemia/reperfusion (I/R) injury is a common pathophysiological phenomenon that is widely present in various primary intestinal diseases and systemic critical illnesses, potentially leading to multiple organ dysfunction and failure, with high morbidity and mortality.

Nature Biomedical Engineering, Published online: 30 April 2026; doi:10.1038/s41551-026-01677-9 STRAIGHT-IN Dual enables allele-specific integration of two DNA constructs into hiPSCs within 1 week. The system was used to programme hiPSCs into distinct cell types and for tunable expression of different transcription factors within the same cell.

Nature Biomedical Engineering, Published online: 30 April 2026; doi:10.1038/s41551-026-01671-1 PRIME-In couples a prime edited donor template with either single or paired genomic nicks to enable precise integration of even large constructs into human cells, as exemplified by high-efficiency integration of a 3-kb CAR construct in T cells.

Nature Biomedical Engineering, Published online: 05 May 2026; doi:10.1038/s41551-026-01672-0 A transferrin receptor-associating polyplex formed by complexing mRNA with modified polyethylenimine enables monocyte trafficking and lymph node targeting for potent and selective cancer vaccination.

Stuart Orkin and Swee Lay Thein shared a Breakthrough Prize in Life Sciences for their research on genetic causes of sickle cell disease and beta-thalassemia that set the stage for approved gene therapies. The treatments are not accessible to everyone, though

Cibus (Nasdaq: CBUS) has transferred gene-edited herbicide-tolerance materials in rice to Latin American partner Interoc, ahead of a forthcoming commercial seed launch. The post Cibus Transfers Gene-Edited Herbicide-Tolerance Traits in Rice to Interoc Ahead of Latin American Launch appeared first on iGrow News .

Patients with exceedingly rare genetic diseases fall through the cracks of the medical system. This doctor is designing drugs for them, one at a time. The post For Every Patient Their Own Drug appeared first on Nautilus .

Three 2026 Breakthrough Prize winners reflect on developing Luxturna, a gene therapy that treats blindness caused by rare inherited eye diseases

Discover how the 2026 healthcare trends are integrating genomics and data to enhance clinical decision-making. The post Trends in 2026 for healthcare – part 3: How are clinical research and genomics creating personalized patient care? appeared first on Capgemini .

Researchers at Johns Hopkins Medicine say they have successfully demonstrated that disrupting an eye structure long suspected of blocking the growth and survival of transplanted nerve cells may help restore vision in people with optic nerve damage. A report on the experiments with animals, stem cells and donated eye tissue was published in Science Translational […]

This is a dose escalation of a dual AAV vector gene therapy for Tay-Sachs and Sandhoff diseases in which a novel bithalamic infusion process was used. The broad distribution of axonal connections of thalamic neurons enables transport of the normal B-hexosaminidase A enzyme throughout the cerebrum.

Russia is developing what officials have described as a “vaccine against aging,” a gene therapy drug aimed at slowing cellular aging by blocking a receptor linked to age-related changes in the body, the deputy science minister said Thursday. Denis Sekirinsky, Russia’s deputy science and higher education minister, said the experimental treatment would target the RAGE […]

For the first time, scientists have shown that heart muscle cells can regrow after a heart attack in humans, a process that had previously only been observed in mice. Groundbreaking work led by specialists from the University of Sydney, the Baird Institute, and the Royal Prince Alfred Hospital in Sydney has found that heart muscle [...]

In a lab room, a toddler, deaf from birth, sits while a tone plays. There’s no reaction. His face does not change.  Six weeks later, after a single injection of an experimental gene therapy, the same toddler is back in the same room. The tone plays. The toddler’s head turns toward the sound. And somewhere […]

Nature Communications, Published online: 02 May 2026; doi:10.1038/s41467-026-72165-6 The tumor suppressor protein p53 is commonly mutated in cancer and has been challenging for therapeutic reactivation. Here, the authors developed a small molecule chemical inducer of proximity to selectively activate p53 transcription and induce cellular senescence and apoptosis in p53Y220C cells.

Gene Therapy, Published online: 30 April 2026; doi:10.1038/s41434-026-00617-1 AAV2 capsid clearance and neuronal trafficking dynamics in the central nervous system

Nature Reviews Drug Discovery, Published online: 01 May 2026; doi:10.1038/d41573-026-00075-9 Dual-vector gene therapy for hearing loss secures first FDA approval

JMIR Publications today featured two new stories in its News and Perspectives section, highlighting a shift toward proactive, tech-integrated health monitoring.