Cystic fibrosis is an inherited genetic disease caused by a single gene mutation in the CFTR protein. Affecting 70,000 people worldwide, this disease is debilitating to patients, resulting in persistent lung infections and decreased quality of life. Due to the various mutation types, there is not currently a single drug...
Nucleic acid therapeutics has cemented itself as one of the most efficacious and robust avenues for treatment of a wide range of diseases from vaccine development to treatment of liver, retina, cancer applications. Non-viral mediated gene delivery using lipid-based nanoparticle (LNP) carriers has proven its compatibility with addressing unexpected, fast...
Messenger RNA (mRNA) therapy has massive potential to treat various genetic disorders by providing effective proteins to the diseased cells and tissues. Its therapeutic applications include protein replacement, genome editing, and immunotherapy. For successful mRNA treatment, mRNA must be delivered to the cytosol of target cells. One of the leading...
Intracellular delivery of mRNA holds great potential for vaccine and therapeutic discovery and development. This is realized by the preeminent role mRNA is playing in the record-breaking pace of SARS-CoV-19 vaccine development. Recent advances in chemical modifications of mRNA have enabled high-efficiency gene expression with superior spatiotemporal control while eliminating...