A novel genetic mitochondrial disorder linked to the LIG3 gene Normally, mitochondrial DNA is repaired and replicated by LIG3 activity, but if the gene contains mutations, enzymes necessary for energy production are not produced, potentially leading to central nervous system symptoms and muscle defects. © Fujita Health University DNA ligase…..
A new agent for brain diseases: mRNA Brain-derived neurotrophic factor mRNA therapeutics for ischemic neuronal death using polyplex nanomicelle. Department of Biofunction Research,TMDU© Brain-derived neurotrophic factor (BDNF) enhances the survival and function of neurons and could serve as a potential candidate neuroprotective agent. However, this protein is too large to…..
First human model for Leigh syndrome [caption id="attachment_9893" align="alignright" width="300"] Human brain organoids with mature neurons in green. ©Dr. Agnieszka Rybak-Wolf[/caption] Leigh syndrome is the most severe mitochondrial disease in children. This orphan disease causes severe muscle weakness, movement defects, and intellectual disabilities. It usually leads to death within the…..
Charcot-Marie Tooth disease: A 100% French RNA-based therapeutic innovation Charcot-Marie Tooth disease is the most common hereditary neurological disease in the world. It affects the peripheral nerves and causes progressive paralysis of the legs and hands. No treatment is currently available to fight this disease, which is due to the…..
Using precision medicine to uncover and treat new skeletal disorders Researchers of the “Cell Biology and Physiology-LABRET” group of the University of Malaga (UMA), together with the Networking Biomedical Research Center in Bioengineering, Biomaterials and Nanomedicine (CIBER-BBN), have described a new genetic skeletal disorder based on a precision medicine strategy.…..
A new gene therapy could pave the way to the treatment of mitochondrial dysfunctions Scientists from Trinity College Dublin have developed a new gene therapy approach that offers promise for one day treating dominant optic atrophy (DOA). This eye disease is characterized by degeneration of the optic nerves and primarily…..
An artificial intelligence approach to create AAV capsids for gene therapies Adeno-associated viruses (AAVs) have become the go-to vehicle for delivering therapeutic gene cargo to target tissues for the recent wave of gene therapies that are in development in academic and biotechnology laboratories (1). However, natural AAVs do not specifically…..
Transforming DNA repair errors into assets A new bioinformatics tool, MHcut, developed by researchers in Kyoto, Japan, and Montreal, Canada, reveals that a natural repair system for DNA damage, microhomology-mediated end joining, is probably far more common in humans than originally assumed. Using MHcut and commercial genome-editing technology, the researchers created…..
Accumulation of DNA mutations found in healthy liver leads to disease New insights into the journey from health to disease in the human liver have been made by scientists at the Wellcome Sanger Institute, Cancer Research UK Cambridge Institute, the University of Cambridge and their collaborators. In the largest study…..
New weapons against superbugs The rapid emergence of antimicrobial resistance is a matter of global concern. According to the WHO, particularly Gram-negative bacteria like Acinetobactebaumannii, Pseudomonas aeruginosa and Enterobacteriaceae that are resistant to the carbapenem and cephalosporin antibiotics, pose a growing threat to human health. These pathogens can cause severe and often life-threatening…..
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