New therapy breakthrough could change the shape of treatment for undruggable diseases For some time, scientists have been working on the major challenge of developing new therapies against many human diseases. Many of these diseases are caused by the abberant action of certain proteins in our cells that are…..
New technology allows molecules to enter cells safely Professor Braeckmans from Ghent University focused the last ten years on a method for safe engineering of therapeutic cells with photothermal nanofibers. Now, Nature Nanotechnology gives insight in how these biocompatible photothermal nanofibers were developed, and how, upon laser irradiation, cells…..
A Z-RNA nanoswitch encoded by "junk DNA" turns-off immune responses against self In a paper published in the May 13th, 2021 issue of PLOS Genetics, Dr. Alan Herbert of InsideOutBio, describes how nature uses a nano-scale Z-RNA switch to turn-off immune responses against self. This Z-RNA nanoswitch sequence, less than…..
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…..
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