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In utero gene editing for monogenic lung disease.
Alapati D, Zacharias WJ, Hartman HA, Rossidis AC, Stratigis JD, Ahn NJ, Coons B, Zhou S, Li H, Singh K, Katzen J, Tomer Y, Chadwick AC, Musunuru K, Beers MF, Morrisey EE, Peranteau WH. Alapati D, et al. Among authors: hartman ha. Sci Transl Med. 2019 Apr 17;11(488):eaav8375. doi: 10.1126/scitranslmed.aav8375. Sci Transl Med. 2019. PMID: 30996081 Free PMC article.
In utero CRISPR-mediated therapeutic editing of metabolic genes.
Rossidis AC, Stratigis JD, Chadwick AC, Hartman HA, Ahn NJ, Li H, Singh K, Coons BE, Li L, Lv W, Zoltick PW, Alapati D, Zacharias W, Jain R, Morrisey EE, Musunuru K, Peranteau WH. Rossidis AC, et al. Among authors: hartman ha. Nat Med. 2018 Oct;24(10):1513-1518. doi: 10.1038/s41591-018-0184-6. Epub 2018 Oct 8. Nat Med. 2018. PMID: 30297903 Free PMC article.
In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease.
Bose SK, White BM, Kashyap MV, Dave A, De Bie FR, Li H, Singh K, Menon P, Wang T, Teerdhala S, Swaminathan V, Hartman HA, Jayachandran S, Chandrasekaran P, Musunuru K, Jain R, Frank DB, Zoltick P, Peranteau WH. Bose SK, et al. Among authors: hartman ha. Nat Commun. 2021 Jul 13;12(1):4291. doi: 10.1038/s41467-021-24443-8. Nat Commun. 2021. PMID: 34257302 Free PMC article.
46 results