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Page 1
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: peranteau wh. 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
Delivery technologies for in utero gene therapy.
Palanki R, Peranteau WH, Mitchell MJ. Palanki R, et al. Among authors: peranteau wh. Adv Drug Deliv Rev. 2021 Feb;169:51-62. doi: 10.1016/j.addr.2020.11.002. Epub 2020 Nov 9. Adv Drug Deliv Rev. 2021. PMID: 33181188 Free PMC article. Review.
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: peranteau wh. Sci Adv. 2021 Jan 13;7(3):eaba1028. doi: 10.1126/sciadv.aba1028. Print 2021 Jan. Sci Adv. 2021. PMID: 33523869 Free PMC article.
Amniotic fluid stabilized lipid nanoparticles for in utero intra-amniotic mRNA delivery.
Swingle KL, Billingsley MM, Bose SK, White B, Palanki R, Dave A, Patel SK, Gong N, Hamilton AG, Alameh MG, Weissman D, Peranteau WH, Mitchell MJ. Swingle KL, et al. Among authors: peranteau wh. J Control Release. 2022 Jan;341:616-633. doi: 10.1016/j.jconrel.2021.10.031. Epub 2021 Nov 3. J Control Release. 2022. PMID: 34742747 Free PMC article.
Foetal genome editing.
Bose SK, Kennedy K, Peranteau WH. Bose SK, et al. Among authors: peranteau wh. Curr Opin Obstet Gynecol. 2023 Apr 1;35(2):134-139. doi: 10.1097/GCO.0000000000000854. Curr Opin Obstet Gynecol. 2023. PMID: 36924409 Free PMC article. Review.
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. Among authors: peranteau wh. 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.
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: peranteau wh. J Clin Invest. 2024 May 9;134(12):e179848. doi: 10.1172/JCI179848. J Clin Invest. 2024. PMID: 38950310 Free PMC article.
InUtero Gene Therapy: Progress and Challenges.
Bose SK, Menon P, Peranteau WH. Bose SK, et al. Among authors: peranteau wh. Trends Mol Med. 2021 Aug;27(8):728-730. doi: 10.1016/j.molmed.2021.05.007. Epub 2021 Jun 24. Trends Mol Med. 2021. PMID: 34176774 Free PMC article. Review.
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: peranteau wh. 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: peranteau wh. Nat Commun. 2021 Jul 13;12(1):4291. doi: 10.1038/s41467-021-24443-8. Nat Commun. 2021. PMID: 34257302 Free PMC article.
143 results