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CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema.
Longhurst HJ, Lindsay K, Petersen RS, Fijen LM, Gurugama P, Maag D, Butler JS, Shah MY, Golden A, Xu Y, Boiselle C, Vogel JD, Abdelhady AM, Maitland ML, McKee MD, Seitzer J, Han BW, Soukamneuth S, Leonard J, Sepp-Lorenzino L, Clark ED, Lebwohl D, Cohn DM. Longhurst HJ, et al. Among authors: sepp lorenzino l. N Engl J Med. 2024 Feb 1;390(5):432-441. doi: 10.1056/NEJMoa2309149. N Engl J Med. 2024. PMID: 38294975 Clinical Trial.
Evaluation of RNAi therapeutics VIR-2218 and ALN-HBV for chronic hepatitis B: Results from randomized clinical trials.
Gane E, Lim YS, Kim JB, Jadhav V, Shen L, Bakardjiev AI, Huang SA, Cathcart AL, Lempp FA, Janas MM, Cloutier DJ, Kaittanis C, Sepp-Lorenzino L, Hinkle G, Taubel J, Haslett P, Milstein S, Anglero-Rodriguez YI, Hebner CM, Pang PS, Yuen MF. Gane E, et al. Among authors: sepp lorenzino l. J Hepatol. 2023 Oct;79(4):924-932. doi: 10.1016/j.jhep.2023.05.023. Epub 2023 Jun 7. J Hepatol. 2023. PMID: 37290591 Free article.
Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy.
Engelbeen S, Pasteuning-Vuhman S, Boertje-van der Meulen J, Parmar R, Charisse K, Sepp-Lorenzino L, Manoharan M, Aartsma-Rus A, van Putten M. Engelbeen S, et al. Among authors: sepp lorenzino l. Nucleic Acid Ther. 2023 Jan;33(1):26-34. doi: 10.1089/nat.2022.0021. Epub 2022 Oct 20. Nucleic Acid Ther. 2023. PMID: 36269327 Free PMC article.
From bench to bedside: Improving the clinical safety of GalNAc-siRNA conjugates using seed-pairing destabilization.
Schlegel MK, Janas MM, Jiang Y, Barry JD, Davis W, Agarwal S, Berman D, Brown CR, Castoreno A, LeBlanc S, Liebow A, Mayo T, Milstein S, Nguyen T, Shulga-Morskaya S, Hyde S, Schofield S, Szeto J, Woods LB, Yilmaz VO, Manoharan M, Egli M, Charissé K, Sepp-Lorenzino L, Haslett P, Fitzgerald K, Jadhav V, Maier MA. Schlegel MK, et al. Among authors: sepp lorenzino l. Nucleic Acids Res. 2022 Jul 8;50(12):6656-6670. doi: 10.1093/nar/gkac539. Nucleic Acids Res. 2022. PMID: 35736224 Free PMC article.
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
Gillmore JD, Gane E, Taubel J, Kao J, Fontana M, Maitland ML, Seitzer J, O'Connell D, Walsh KR, Wood K, Phillips J, Xu Y, Amaral A, Boyd AP, Cehelsky JE, McKee MD, Schiermeier A, Harari O, Murphy A, Kyratsous CA, Zambrowicz B, Soltys R, Gutstein DE, Leonard J, Sepp-Lorenzino L, Lebwohl D. Gillmore JD, et al. Among authors: sepp lorenzino l. N Engl J Med. 2021 Aug 5;385(6):493-502. doi: 10.1056/NEJMoa2107454. Epub 2021 Jun 26. N Engl J Med. 2021. PMID: 34215024 Clinical Trial.
Centyrin ligands for extrahepatic delivery of siRNA.
Klein D, Goldberg S, Theile CS, Dambra R, Haskell K, Kuhar E, Lin T, Parmar R, Manoharan M, Richter M, Wu M, Mendrola Zarazowski J, Jadhav V, Maier MA, Sepp-Lorenzino L, O'Neil K, Dudkin V. Klein D, et al. Among authors: sepp lorenzino l. Mol Ther. 2021 Jun 2;29(6):2053-2066. doi: 10.1016/j.ymthe.2021.02.015. Epub 2021 Feb 15. Mol Ther. 2021. PMID: 33601052 Free PMC article.
Knockdown of Virus Antigen Expression Increases Therapeutic Vaccine Efficacy in High-Titer Hepatitis B Virus Carrier Mice.
Michler T, Kosinska AD, Festag J, Bunse T, Su J, Ringelhan M, Imhof H, Grimm D, Steiger K, Mogler C, Heikenwalder M, Michel ML, Guzman CA, Milstein S, Sepp-Lorenzino L, Knolle P, Protzer U. Michler T, et al. Among authors: sepp lorenzino l. Gastroenterology. 2020 May;158(6):1762-1775.e9. doi: 10.1053/j.gastro.2020.01.032. Epub 2020 Jan 28. Gastroenterology. 2020. PMID: 32001321
Safety evaluation of 2'-deoxy-2'-fluoro nucleotides in GalNAc-siRNA conjugates.
Janas MM, Zlatev I, Liu J, Jiang Y, Barros SA, Sutherland JE, Davis WP, Liu J, Brown CR, Liu X, Schlegel MK, Blair L, Zhang X, Das B, Tran C, Aluri K, Li J, Agarwal S, Indrakanti R, Charisse K, Nair J, Matsuda S, Rajeev KG, Zimmermann T, Sepp-Lorenzino L, Xu Y, Akinc A, Fitzgerald K, Vaishnaw AK, Smith PF, Manoharan M, Jadhav V, Wu JT, Maier MA. Janas MM, et al. Among authors: sepp lorenzino l. Nucleic Acids Res. 2019 Apr 23;47(7):3306-3320. doi: 10.1093/nar/gkz140. Nucleic Acids Res. 2019. PMID: 30820542 Free PMC article.
83 results