First vascularized model of stem cell islet cells [caption id="attachment_11877" align="aligncenter" width="1024"] Engineered vasculature induces functional maturation of pluripotent stem cell-derived islet organoidsResearchers have devised the right conditions to grow vascularized stem cell islets. The image shows vasculature (red) tightly wrapped around insulin-producing cells (green) in the islets (blue). ©…..
Why are patients with mitochondrial disease more susceptible to infections? The cell at the top right is dying through caspase-11 dependent pyroptosis, the mechanism West and colleagues identified in this paper. It's mitochondria are gone and the nucleus also condensed. ©The Jackson Laboratory Patients with rare mitochondrial diseases already face a…..
GNT0004 product confirms its clinical efficacy with stabilization of motor functions in patients treated at the effective dose for up to 2 years Genethon has unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne Muscular Dystrophy (GNT-016-MDYF) at the annual meeting of the American Society…..
Study suggests new molecular strategy for treating fragile X syndrome Building on more than two decades of research, a study by MIT neuroscientists at The Picower Institute for Learning and Memory reports a new way to treat pathology and symptoms of fragile X syndrome, the most common genetically-caused autism spectrum…..
Novel gene therapy trial for sickle cell disease launches in the US The study uses CRISPR to correct sickle cell mutation and aims to free patients of the disease. UCSF Benioff Children's Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease. The…..
Preclinical data for AAV-FGF21 gene therapy shows durable reversal of liver fibrosis UAB researchers have cured liver disease MASH in mouse models by a one-time gene therapy . The results were obtained with a single intramuscular administration of the therapeutic viral vectors AAV-FGF21. The research has also determined that most…..
An epigenome editing toolkit to dissect the mechanisms of gene regulation A study from the Hackett group at EMBL Rome led to the development of a powerful epigenetic editing technology, which unlocks the ability to precisely program chromatin modifications. Creative depiction of the epigenetic editing toolkit: each building represents the…..
New approach to single-cell RNA structure sequencing unveils biomarkers for human development and disease Different cells have different RNA structures and studying those structures offers insights into cell functions. © A*STAR’s GIS Researchers from A*STAR’s Genome Institute of Singapore (GIS) have found an innovative approach to sequencing single-cell RNA to…..
NIH study shows how genes in retina get regulated during development Genome topology map of human retina development lays foundation for understanding diverse clinical phenotypes in simple and complex eye diseases [caption id="" align="aligncenter" width="555"] This image metaphorically captures the cell differentiation process during retinal organoid development. Loose yarn representing…..
Fragile X syndrome begins developing even before birth Fragile X syndrome (FXS), the most common form of inherited intellectual disability, may be unfolding in brain cells even before birth, despite typically going undiagnosed until age 3 or later. These results could fundamentally change how scientists understand the developmental origins of…..
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