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…..
DNA Script Partners with Moderna to Develop On‐Demand Vaccines and Therapeutics for DARPA DNA Script, a disruptive DNA synthesis company engineering biology to accelerate breakthroughs in life science, is partnering with Moderna to develop a prototype for rapid mobile manufacturing of vaccines and therapeutics as part of the Defense Advanced…..
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…..
Evonik delivers first lipids from German facility to BioNTech The German company Evonik is delivering first batches of urgently needed lipids used in production of COVID-19 vaccine to BioNTech on April 22. Initially, delivery was scheduled to start in the middle of the year. But, specialists at Evonik’s Hanau site…..
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…..
New study expands evaluation of gene therapy for spinal muscular atrophy German researchers have just published results of a retrospective analysis of routine data collected from all patients with spinal muscular atrophy (SMA) treated with Zolgensma® (onasemnogene abeparvovec - adeno-associated viral (AAV9) vector-based gene therapy that introduces a functional copy…..
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…..
Celonic will manufacture over 100 million doses of Curevac's COVID-19 Vaccine Candidate, CVnCoV CureVac and Celonic Group have conclud a partnership for the production of CureVac’s mRNA-based COVID-19 vaccine candidate, CVnCoV. CureVac began development of mRNA-based COVID-19 vaccine candidates in January 2020 and CVnCoV is the vaccine candidate chosen for…..
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…..