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Efficient and safe therapeutic use of paired Cas9-nickases for primary hyperoxaluria type 1.
Torella L, Klermund J, Bilbao-Arribas M, Tamayo I, Andrieux G, Chmielewski KO, Vales A, Olagüe C, Moreno-Luqui D, Raimondi I, Abad A, Torrens-Baile J, Salido E, Huarte M, Hernaez M, Boerries M, Cathomen T, Zabaleta N, Gonzalez-Aseguinolaza G. Torella L, et al. Among authors: zabaleta n. EMBO Mol Med. 2024 Jan;16(1):112-131. doi: 10.1038/s44321-023-00008-8. Epub 2024 Jan 5. EMBO Mol Med. 2024. PMID: 38182795 Free PMC article.
Tracing the fate of AAV vectors in the body.
Zabaleta N, Gil-Farina I. Zabaleta N, et al. Nat Biotechnol. 2023 Nov 27. doi: 10.1038/s41587-023-02047-5. Online ahead of print. Nat Biotechnol. 2023. PMID: 38012451 No abstract available.
Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice.
Reichart D, Newby GA, Wakimoto H, Lun M, Gorham JM, Curran JJ, Raguram A, DeLaughter DM, Conner DA, Marsiglia JDC, Kohli S, Chmatal L, Page DC, Zabaleta N, Vandenberghe L, Liu DR, Seidman JG, Seidman C. Reichart D, et al. Among authors: zabaleta n. Nat Med. 2023 Feb;29(2):412-421. doi: 10.1038/s41591-022-02190-7. Epub 2023 Feb 16. Nat Med. 2023. PMID: 36797483 Free PMC article.
Gene therapy for liver diseases - progress and challenges.
Zabaleta N, Unzu C, Weber ND, Gonzalez-Aseguinolaza G. Zabaleta N, et al. Nat Rev Gastroenterol Hepatol. 2023 May;20(5):288-305. doi: 10.1038/s41575-022-00729-0. Epub 2023 Jan 16. Nat Rev Gastroenterol Hepatol. 2023. PMID: 36646909 Review.
Ancestral library identifies conserved reprogrammable liver motif on AAV capsid.
Zinn E, Unzu C, Schmit PF, Turunen HT, Zabaleta N, Sanmiguel J, Fieldsend A, Bhatt U, Diop C, Merkel E, Gurrala R, Peacker B, Rios C, Messemer K, Santos J, Estelien R, Andres-Mateos E, Wagers AJ, Tipper C, Vandenberghe LH. Zinn E, et al. Among authors: zabaleta n. Cell Rep Med. 2022 Nov 15;3(11):100803. doi: 10.1016/j.xcrm.2022.100803. Epub 2022 Nov 2. Cell Rep Med. 2022. PMID: 36327973 Free PMC article.
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques.
Zabaleta N, Bhatt U, Hérate C, Maisonnasse P, Sanmiguel J, Diop C, Castore S, Estelien R, Li D, Dereuddre-Bosquet N, Cavarelli M, Gallouët AS, Pascal Q, Naninck T, Kahlaoui N, Lemaitre J, Relouzat F, Ronzitti G, Thibaut HJ, Montomoli E, Wilson JM, Le Grand R, Vandenberghe LH. Zabaleta N, et al. Mol Ther. 2022 Sep 7;30(9):2952-2967. doi: 10.1016/j.ymthe.2022.05.007. Epub 2022 May 10. Mol Ther. 2022. PMID: 35546782 Free PMC article.
Computed tomography and [18F]-FDG PET imaging provide additional readouts for COVID-19 pathogenesis and therapies evaluation in non-human primates.
Naninck T, Kahlaoui N, Lemaitre J, Maisonnasse P, De Mori A, Pascal Q, Contreras V, Marlin R, Relouzat F, Delache B, Hérate C, Aldon Y, van Gils M, Zabaleta N, Ho Tsong Fang R, Bosquet N, Sanders RW, Vandenberghe LH, Chapon C, Le Grand R. Naninck T, et al. Among authors: zabaleta n. iScience. 2022 Apr 15;25(4):104101. doi: 10.1016/j.isci.2022.104101. Epub 2022 Mar 17. iScience. 2022. PMID: 35313622 Free PMC article.
24 results