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We developed Twin Selective Organ-Concentrating on Lipid Nanoparticles (Twin SORT LNPs) able to delivering base editors to a number of organs. Base editor Twin SORT LNPs corrected disease-causing mutations in each the liver and lungs, the first organs affected in alpha-1 antitrypsin deficiency.
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References
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Strnad, P., McElvaney, N. G. & Lomas, D. A. Alpha1-antitrypsin deficiency. N. Engl. J. Med. 382, 1443–1455 (2020). A assessment article on AATD.
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Zamora, M. R. & Ataya, A. Lung and liver transplantation in sufferers with alpha-1 antitrypsin deficiency. Ther. Adv. Continual Dis. 12, (Suppl.) 20406223211002988 (2021). A assessment article that presents present therapy choices for AATD.
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Stiles, Ok. M. et al. Intrapleural gene remedy for alpha-1 antitrypsin deficiency-related lung illness. Continual Obstr. Pulm. Dis. 5, 244–257 (2018). A assessment article on gene remedy for AATD.
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Cheng, Q. et al. Selective organ focusing on (SORT) nanoparticles for tissue-specific mRNA supply and CRISPR-Cas gene modifying. Nat. Nanotechnol. 15, 313–320 (2020). This paper reviews the LNP SORT methodology.
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Beam Therapeutics. A part 1/2 dose-exploration and dose-expansion research to judge the security and efficacy of BEAM-302 in grownup sufferers with alpha-1 antitrypsin deficiency (AATD)-associated lung illness and/or liver illness. https://clinicaltrials.gov/study/NCT06389877 (2025). A scientific research that presents base editor AATD therapy.
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This can be a abstract of: Kim, M. et al. Twin SORT LNPs for multi-organ base modifying. Nat. Biotechnol. https://doi.org/10.1038/s41587-025-02675-z (2025).
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Delivering base editors to the liver and lungs in alpha-1 antitrypsin deficiency.
Nat Biotechnol (2025). https://doi.org/10.1038/s41587-025-02705-w
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DOI: https://doi.org/10.1038/s41587-025-02705-w
