Search results for life+sciences+%3e+therapeutics+%3e+gene+editing

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Beta-sitosterol lipid nanoparticles for aerosolized gene editing in lung diseases
Background Cystic fibrosis (CF) is a life-threatening genetic disorder caused by mutations in the CFTR gene, leading to progressive lung dysfunction. A particularly challenging mutation, W1282X, introduces a premature stop codon that results in non-functional CFTR protein. Patients with this mutation are largely unresponsive to current small molecule...
Improving the microbiome health of bees
Genetically engineered bacteria from the microbiome of bees are used to produce RNA interference (RNAi) molecules and other genetic tools to target and downregulate specific genes in bees or their pathogens. By reducing pathogen loads and altering gene expression of bees, this technology can combat threats to bee population, like colony collapse disorder,...
Fusion protein technology for site‑specific integration of RNA into DNA genomes
The technology integrates target RNA into DNA genomes by adding short DNA tails to RNA. Direct RNA integration into CRISPR arrays eliminates the use of harsh transfection methods that fragment DNA sequences. Background The development of CRISPR-Cas systems, which are widely used for precise genetic modifications, has advanced the field of genome...