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CAR T cells produced in vivo to treat cardiac injury.
Rurik JG, Tombácz I, Yadegari A, Méndez Fernández PO, Shewale SV, Li L, Kimura T, Soliman OY, Papp TE, Tam YK, Mui BL, Albelda SM, Puré E, June CH, Aghajanian H, Weissman D, Parhiz H, Epstein JA. Rurik JG, et al. Science. 2022 Jan 7;375(6576):91-96. doi: 10.1126/science.abm0594. Epub 2022 Jan 6. Science. 2022. PMID: 34990237 Free PMC article.
In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels.
Rothgangl T, Dennis MK, Lin PJC, Oka R, Witzigmann D, Villiger L, Qi W, Hruzova M, Kissling L, Lenggenhager D, Borrelli C, Egli S, Frey N, Bakker N, Walker JA 2nd, Kadina AP, Victorov DV, Pacesa M, Kreutzer S, Kontarakis Z, Moor A, Jinek M, Weissman D, Stoffel M, van Boxtel R, Holden K, Pardi N, Thöny B, Häberle J, Tam YK, Semple SC, Schwank G. Rothgangl T, et al. Nat Biotechnol. 2021 Aug;39(8):949-957. doi: 10.1038/s41587-021-00933-4. Epub 2021 May 19. Nat Biotechnol. 2021. PMID: 34012094 Free PMC article.
PECAM-1 directed re-targeting of exogenous mRNA providing two orders of magnitude enhancement of vascular delivery and expression in lungs independent of apolipoprotein E-mediated uptake.
Parhiz H, Shuvaev VV, Pardi N, Khoshnejad M, Kiseleva RY, Brenner JS, Uhler T, Tuyishime S, Mui BL, Tam YK, Madden TD, Hope MJ, Weissman D, Muzykantov VR. Parhiz H, et al. J Control Release. 2018 Dec 10;291:106-115. doi: 10.1016/j.jconrel.2018.10.015. Epub 2018 Oct 15. J Control Release. 2018. PMID: 30336167 Free PMC article.