New Article Published in Science Highlights the Potential of ReCode Therapeutics’ Systemic Lung Delivery and CFTR Gene Correction
ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced the publication of a research article in Science.
- ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today announced the publication of a research article in Science.
- The article titled " In vivo editing of lung stem cells for durable gene correction in mice " describes the ability of Selective Organ Targeting (SORT) lipid nanoparticles (LNPs) to facilitate high levels of persistent gene editing in lung stem cells when given systemically via intravenous injection.
- This is the first study to show that LNPs carrying gene editing tools can effectively correct a gene in lung stem cells when administered intravenously and delivered through systemic circulation.
- Proof of concept was demonstrated for correction of the CF mutation CFTR R553X, which is untreatable with currently available small molecule therapies.