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In utero delivery of targeted ionizable lipid nanoparticles facilitates in vivo gene editing of hematopoietic stem cells.
Palanki R, Riley JS, Bose SK, Luks V, Dave A, Kus N, White BM, Ricciardi AS, Swingle KL, Xue L, Sung D, Thatte AS, Safford HC, Chaluvadi VS, Carpenter M, Han EL, Maganti R, Hamilton AG, Mrksich K, Billingsley MB, Zoltick PW, Alameh MG, Weissman D, Mitchell MJ, Peranteau WH. Palanki R, et al. Among authors: zoltick pw. Proc Natl Acad Sci U S A. 2024 Aug 6;121(32):e2400783121. doi: 10.1073/pnas.2400783121. Epub 2024 Jul 30. Proc Natl Acad Sci U S A. 2024. PMID: 39078677
Ionizable lipid nanoparticles for in utero mRNA delivery.
Riley RS, Kashyap MV, Billingsley MM, White B, Alameh MG, Bose SK, Zoltick PW, Li H, Zhang R, Cheng AY, Weissman D, Peranteau WH, Mitchell MJ. Riley RS, et al. Among authors: zoltick pw. Sci Adv. 2021 Jan 13;7(3):eaba1028. doi: 10.1126/sciadv.aba1028. Print 2021 Jan. Sci Adv. 2021. PMID: 33523869 Free PMC article.
Preexisting maternal immunity to AAV but not Cas9 impairs in utero gene editing in mice.
Riley JS, Luks VL, Berkowitz CL, Dumitru AM, Kus NJ, Dave A, Menon P, De Paepe ME, Jain R, Li L, Dugoff L, Teefey CP, Alameh MG, Zoltick PW, Peranteau WH. Riley JS, et al. Among authors: zoltick pw. J Clin Invest. 2024 May 9;134(12):e179848. doi: 10.1172/JCI179848. J Clin Invest. 2024. PMID: 38950310 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: zoltick pw. 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.
60 results