Fig. 2
From: Modified mRNA as a new therapeutic option for pediatric respiratory diseases and hemoglobinopathies
SNP correction using nec-mNRA and ssODNs. To facilitate site-specific gene correction of single nucleotide polymorphisms (SNPs), nec-mRNA and single-stranded oligodeoxynucleotides (ssODNs) can be efficiently administered to target cells (e.g., lung cells) by complexing both into positively charged nanoparticles for enhanced cellular uptake. Once in the cell, nec-mRNA gets translated into a functional nuclease that subsequently translocates to the nucleus and binds at sequence-specific sites next to the SNP. Generating a double-strand break (DSB), the genomic mutation can be corrected in the presence of ssODNs by means of homology-directed repair, resulting in proper gene function