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232 results

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Improved specificity and efficacy of base-editing therapies with hybrid guide RNAs.
Whittaker MN, Brooks DL, Quigley A, Jindal I, Said H, Qu P, Wang JZ, Ahrens-Nicklas RC, Musunuru K, Alameh MG, Peranteau WH, Wang X. Whittaker MN, et al. Among authors: musunuru k. bioRxiv [Preprint]. 2024 Jun 16:2024.04.22.590531. doi: 10.1101/2024.04.22.590531. bioRxiv. 2024. PMID: 38712058 Free PMC article. Preprint.
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: musunuru k. 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 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: musunuru k. Sci Transl Med. 2019 Apr 17;11(488):eaav8375. doi: 10.1126/scitranslmed.aav8375. Sci Transl Med. 2019. PMID: 30996081 Free PMC article.
Angiopoietin-Like 3: From Discovery to Therapeutic Gene Editing.
Wang X, Musunuru K. Wang X, et al. Among authors: musunuru k. JACC Basic Transl Sci. 2019 Oct 28;4(6):755-762. doi: 10.1016/j.jacbts.2019.05.008. eCollection 2019 Oct. JACC Basic Transl Sci. 2019. PMID: 31709322 Free PMC article. Review.
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: musunuru k. Nat Commun. 2021 Jul 13;12(1):4291. doi: 10.1038/s41467-021-24443-8. Nat Commun. 2021. PMID: 34257302 Free PMC article.
Therapeutic application of genome editing in dyslipidemia.
Whittaker MN, Musunuru K. Whittaker MN, et al. Among authors: musunuru k. Curr Opin Lipidol. 2022 Apr 1;33(2):133-138. doi: 10.1097/MOL.0000000000000805. Curr Opin Lipidol. 2022. PMID: 34907967 Review.
232 results