gene-therapy

This Collection highlights recent progress in genome editing technology developments. These developments offer exciting opportunities to reshape our approach to understanding biology, driving progress in various fields such as gene therapy, medicine, and agriculture.

Nature Medicine, Published online: 16 June 2026; doi:10.1038/d41591-026-00031-5 Engineered heart muscle allografts derived from induced pluripotent stem cells show promising early outcomes in patients with treatment-refractory advanced heart failure with reduced left ventricular ejection fraction, in support of further clinical investigation.

Samagya Banskota grew up in a family of doctors in Nepal, aware from a young age of how physicians could change people’s lives. Today she is a biomedical engineer at Boston University, studying ways to create safer, more effective therapies for a multitude of diseases. Banskota is creating so-called viruslike particles to deliver gene-editing technologies to cells. These engineered particles have…

Medical researchers can spend their entire careers hunting for genetic clues to explain the cellular dysfunction behind diseases. These quests can have huge payoffs: gene therapies that target a disease’s key drivers. Christina V. Theodoris wondered whether an artificial-intelligence model could complete this task not only more quickly than previous approaches but across multiple targets and dise…

Explore how viral vector CDMOs improve late-stage cell and gene therapy success by reducing risk, variability, and complexity. The post Viral vector production: How CDMOs influence late-stage development success appeared first on Labiotech.eu . © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/o…

(MIT Technology Review) – Earlier this week, Life Biosciences, a biotech company focused on reversing age-related diseases, announced that it had dosed its first volunteer. A person with glaucoma has had an experimental treatment injected straight into their eyeball. The … Read More

(Nature) – A participant in a landmark clinical trial has been given a cellular-reprogramming treatment that aims to rejuvenate damaged cells in the eye. Test time has arrived: the first person has been treated in a highly anticipated gene-therapy trial … Read More

Deep brain stimulation (DBS) has been used for more than three decades to treat motor symptoms of Parkinson’s disease. Today, more than 200,000 patients worldwide have been implanted with these systems, which continuously deliver electrical stimulation to specific deep-brain regions to reduce rigidity and tremor. Yet despite its clinical success, conventional deep brain stimulation remains [&#823…

University of Maryland School of Medicine (UMSOM) Researchers have identified a target that may improve the response of CAR T-cell therapy, a treatment for patients with recurrent or difficult to treat blood cancers.

Dual-target CAR T cell therapy for recurrent glioblastoma (GBM), delivered directly into the cerebrospinal fluid (CSF), triggers a broad immune response, with natural killer (NK) cell activation linked to better patient outcomes and longer overall survival.

World Sickle Cell Day, observed annually on June 19th, raises global awareness about sickle cell disease (SCD): a hereditary blood disorder affecting millions worldwide that causes red blood cells to deform into a rigid, sickle shape, disrupting oxygen flow and causing severe pain, organ damage, and life-threatening complications. About 1 in 13 African Americans carry the sickle cell trait, and m…

Deep brain stimulation (DBS) has been used for more than three decades to treat motor symptoms of Parkinson's disease.

A research team from the Universitat Autònoma de Barcelona (UAB) has shown that a one-time administration of a gene therapy expressing the metabolic factor FGF21 can prolong health span in old mice.

Plant scientists are searching for faster, more flexible ways to rewrite crop genomes without relying on slow and difficult transformation pipelines. A new study shows that plant viruses from the potyvirus group can act as delivery vehicles for genome-editing instructions in plants that express Cas12a.

Every single day, human cells are forced to contend with genomic DNA damage. Caused by everything from environmental toxins and sunlight radiation to the simple, everyday byproduct of our own respiration and metabolism, this stress gradually wears down our telomeres.

Nature Communications, Published online: 15 June 2026; doi:10.1038/s41467-026-74435-9 Breast cancers with HER2 overexpression can vary in whether they also express the estrogen receptor, influencing tumor behavior and treatment response. Here, the authors show that in transgenic mouse models, the HER2Δ16 splice variant promotes the development of aggressive luminal tumors by facilitating luminal …

Boston-based biotechnology company Life Biosciences has launched the first-in-human clinical trials of a pioneering “partial cellular reprogramming” technique designed to treat optic nerve damage caused by glaucoma and NAION. Based on previous genetic research, the therapy utilizes a modified virus to deliver three youth-restoring genes to retinal cells, aiming to reverse cellular aging while pre…

The first participant has been treated in a landmark clinical trial of cellular reprogramming, which aims to rejuvenate aging cells

Researchers from Amsterdam UMC, in collaboration with other hospitals, have successfully completed the first-ever Phase 3 study of an in vivo CRISPR therapy.

Revolutionary devices, age rewinding, and cell regeneration: scientists are finding multiple promising approaches for curing blindness, and even for stopping vision loss before it starts.