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Bypassing broken genes

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 new agent for brain diseases: mRNA

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

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…..