codon-optimized elastin gene therapy

Codon-optimized elastin gene therapy is a therapeutic approach designed to restore functional elastin production and elastic fiber assembly in tissues affected by genetic or acquired elastinopathies. This strategy involves the delivery of a human elastin (ELN) gene sequence that has been modified to optimize codon usage, thereby overcoming translational limitations associated with rare codons in the native sequence. A specific implementation of this technology, known as **Opti-ELN**, utilizes either plasmid DNA or nucleotide-modified mRNA encapsulated in lipid nanoparticles (LNPs). Developed by researchers at Nationwide Children's Hospital and The Ohio State University, Opti-ELN has demonstrated the ability to significantly increase elastin secretion and organize elastic fibers in human cell lines, including fibroblasts from Williams syndrome patients. Preclinical studies in mouse models have shown successful human elastin expression in skin, lung, and vascular tissues following subcutaneous or systemic administration.

Other names

Opti-ELN pDNAOpti-ELN mRNA-LNP