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A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing.
Finn JD, Smith AR, Patel MC, Shaw L, Youniss MR, van Heteren J, Dirstine T, Ciullo C, Lescarbeau R, Seitzer J, Shah RR, Shah A, Ling D, Growe J, Pink M, Rohde E, Wood KM, Salomon WE, Harrington WF, Dombrowski C, Strapps WR, Chang Y, Morrissey DV. Finn JD, et al. Among authors: seitzer j. Cell Rep. 2018 Feb 27;22(9):2227-2235. doi: 10.1016/j.celrep.2018.02.014. Cell Rep. 2018. PMID: 29490262 Free 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: seitzer j. 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.
siRNA-mediated knockdown of P450 oxidoreductase in rats: a tool to reduce metabolism by CYPs and increase exposure of high clearance compounds.
Burke RS, Somasuntharam I, Rearden P, Brown D, Deshmukh SV, DiPietro MA, DiMuzio J, Eisenhandler R, Fauty SE, Gibson C, Gindy ME, Hamilton KA, Knemeyer I, Koeplinger KA, Kwon HW, Lifsted TQ, Menzel K, Patel M, Pudvah N, Rudd DJ, Seitzer J, Strapps WR, Prueksaritanont T, Thompson CD, Hochman JH, Carr BA. Burke RS, et al. Among authors: seitzer j. Pharm Res. 2014 Dec;31(12):3445-60. doi: 10.1007/s11095-014-1433-0. Epub 2014 Jul 1. Pharm Res. 2014. PMID: 24980206
Quantitative evaluation of siRNA delivery in vivo.
Pei Y, Hancock PJ, Zhang H, Bartz R, Cherrin C, Innocent N, Pomerantz CJ, Seitzer J, Koser ML, Abrams MT, Xu Y, Kuklin NA, Burke PA, Sachs AB, Sepp-Lorenzino L, Barnett SF. Pei Y, et al. Among authors: seitzer j. RNA. 2010 Dec;16(12):2553-63. doi: 10.1261/rna.2255810. Epub 2010 Oct 12. RNA. 2010. PMID: 20940339 Free PMC article.
2023 White Paper on Recent Issues in Bioanalysis: ISR for ADA Assays, the Rise of dPCR vs qPCR, International Reference Standards for Vaccine Assays, Anti-AAV TAb Post-Dose Assessment, NanoString Validation, ELISpot as Gold Standard (Part 3 - Recommendations on Gene Therapy, Cell Therapy, Vaccines Immunogenicity & Technologies; Biotherapeutics Immunogenicity & Risk Assessment; ADA/NAb Assay/Reporting Harmonization).
Mora J, Palmer R, Wagner L, Wu B, Partridge M, Meena, Sonderegger I, Smeraglia J, Bivi N, Dakappagari N, Diebold S, Garofolo F, Grimaldi C, Kalina W, Kamerud J, Kar S, Marshall JC, Mayer C, Melton A, Merdek K, Nolan K, Picard S, Shao W, Seitzer J, Tanaka Y, Tounekti O, Vigil A, Walravens K, Xu J, Xu W, Xu Y, Yang L, Zhu L, Verthelyi D, Kubiak RJ, Coble K, Gupta S, Abhari MR, Richards S, Song Y, Ullmann M, Calderon B, Cludts I, Gunn GR, Gupta S, Ishii-Watabe A, Manangeeswaran M, Maxfield K, McCush F, O'Day C, Peng K, Poetzl J, Rasamoelisolo M, Saad OM, Scheibner K, Shubow S, Song S, Thacker S. Mora J, et al. Among authors: seitzer j. Bioanalysis. 2024 Apr;16(7):77-119. doi: 10.4155/bio-2024-0024. Epub 2024 Feb 22. Bioanalysis. 2024. PMID: 38389403 Free article.
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: seitzer j. N Engl J Med. 2024 Feb 1;390(5):432-441. doi: 10.1056/NEJMoa2309149. N Engl J Med. 2024. PMID: 38294975 Clinical Trial.
11 results