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genome editing using 244- LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice. | LitMetric

genome editing using 244- LNPs and low-dose AAV achieves therapeutic threshold in hemophilia A mice.

Mol Ther Nucleic Acids

Graduate School of International Agricultural Technology and Institute of Green BioScience and Technology, Seoul National University, Pyeongchang, Gangwon 25354, Korea.

Published: December 2023

Gene therapy and rebalancing therapy have emerged as promising approaches for treating hemophilia A, but there are limitations, such as temporary efficacy due to individual differences. Genome editing for hemophilia has shown long-term therapeutic potential in preclinical trials. However, a cautious approach is necessary because genome editing is irreversible. Therefore, we attempted to induce low-level human factor 8 (hF8) gene knockin (KI) using 244- lipid nanoparticles and low-dose adeno-associated virus to minimize side effects and achieve a therapeutic threshold in hemophilia A mice. We selected the serpin family C member 1, , locus as a target to enable a combined rebalancing strategy with h KI to augment efficacy. This strategy improved blood coagulation activity and reduced hemophilic complications without adverse effects. Furthermore, hemophilic mice with genome editing exhibit enhanced survival for 40 weeks. Here, we demonstrate an effective, safe, and sustainable treatment for hemophilia A. This study provides valuable information to establish safe and long-term genome-editing-mediated treatment strategies for treating hemophilia and other protein-deficient genetic diseases.

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Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10616378PMC
http://dx.doi.org/10.1016/j.omtn.2023.102050DOI Listing

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