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…..
Drug discovery on an unprecedented scale [caption id="" align="aligncenter" width="700"] Artistic impression of artificial intelligence in drug design - ©Ina Pöhner[/caption] Boosting virtual screening with machine learning allowed for a 10-fold time reduction in the processing of 1.56 billion drug-like molecules. Researchers from the University of Eastern Finland teamed up with industry…..
Transposons: From ‘selfish’ genes to key elements in biological processes A new SISSA and IIT study reveals unexpected functionality of LINE transposons, paving new paths in the search for cures for neurodevelopmental and ageing disorders. Double Helix generated by AI - ©Remo Sanges In 2001 the sequencing of the…..
NIH clinical trial of universal flu vaccine candidate begins A colorized transmission electron micrograph of influenza A virus particles, colorized orange, isolated from a patient sample and then propagated in cell culture. ©NIAID Enrollment in a Phase 1 trial of a new investigational universal influenza vaccine candidate has begun at…..
Application of base editors in organoids opens new doors for cancer research The development of new cancer treatments depends on suitable tumor models in the laboratory. Researchers from the Organoid group (Hubrecht Institute) and the Princess Máxima Center have used base editors to simultaneously introduce multiple cancer-related mutations into healthy…..
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