Prime editing corrects a childhood genetic disease in mice
 Alternating hemiplegia of childhood (AHC) is a rare, one-in-a-million genetic disease that affects kids. Most cases are…
Breakthrough gene therapy reverses genetic deafness across age groups
Using gene therapy to treat hereditary deafness is safe and effective in both children and adults, according to…
Targeted in vivo gene integration of a secretion-enabled GLP-1 receptor agonist reverses diet-induced non-genetic obesity and pre-diabetes
Plasmid construction The Exe4 gene was codon-optimized using the GeneSmart codon optimization tool (https://www.genscript.com/gensmart-free-gene-codon-optimization.html) based on the amino…
Rescued by fat bubbles: Scientists treat rare genetic disease with designer molecule | Lifestyle
Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with…