Novel nanotech improves cystic fibrosis antibiotic by 100,000-fold Cystic fibrosis is causing severe impairments to a person's lungs, airways and digestive system, trapping bacteria and leading to recurrent infections. Lung failure is the major cause of death for people with this disease. World-first nanotechnology developed by the University of South…..
New research on why mutations in a gene leads to mitochondrial disease Image of mitochondria (yellow-green) around the cell nucleus (blue). ©Dr Matthew Eramo Mitochondrial diseases are inherited, chronic illnesses that can present at birth or develop later in life and occurs when mitochondria fail to produce enough energy for…..
Bypassing broken genes Researchers at Penn State have developed a new approach to gene editing using the CRISPR/Cas9 system bypasses disease-causing mutations in a gene, enabling treatment of genetic diseases linked to a single gene, such as cystic fibrosis, certain types of sickle cell anemia, and other rare diseases. The…..
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…..