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…..
Merck KGaA to triple its ADC manufacturing capacity in St. Louis The German science and technology group Merck KGaA has announced a € 70 million expansion of its antibody-drug conjugates (ADC) manufacturing capabilities and capacity at its Bioconjugation Center of Excellence facility in St. Louis, Missouri, USA. This investment…..
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…..
Vivet Therapeutics receives €4.9 million to advance development of a gene therapy for the treatment of cerebrotendinous xanthomatosis Funding from French Government as part of the France Health Innovation Plan 2030 Vivet Therapeutics, a French clinical stage biotech company developing novel and long-lasting gene therapies for rare inherited metabolic disorders,…..
AiCuris and the German Center for Infection Research (DZIF) sign Collaboration and License Option Agreement ©DZIF AiCuris Anti-infective Cures AG and the German Center for Infection Research (DZIF) have signed a collaboration and license option agreement to identify novel targets for antiviral therapy and to discover new active agents for…..
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…..
Using artificial intelligence to discover therapeutic antibodies Former EMBL staff scientist founds a start-up – DenovAI – for broader, faster and cheaper antibody discovery using advanced machine learning and computational biophysics. The EMBL scientists who developed the AI-driven technology to design proteins that DenovAI will harness. From left to right:…..
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…..
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