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Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease.
Palanki R, Bose SK, Dave A, White BM, Berkowitz C, Luks V, Yaqoob F, Han E, Swingle KL, Menon P, Hodgson E, Biswas A, Billingsley MM, Li L, Yiping F, Carpenter M, Trokhan A, Yeo J, Johana N, Wan TY, Alameh MG, Bennett FC, Storm PB, Jain R, Chan J, Weissman D, Mitchell MJ, Peranteau WH. Palanki R, et al. ACS Nano. 2023 Jul 25;17(14):13594-13610. doi: 10.1021/acsnano.3c02268. Epub 2023 Jul 17. ACS Nano. 2023. PMID: 37458484 Free PMC article.
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. 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