Al Musaimi O, Al Shaer D, Albericio F, de la Torre BG. 2020 FDA TIDES (Peptides and Oligonucleotides) Harvest. Pharmaceuticals. 2021;14(2):145.
Article PubMed PubMed Central Google Scholar
Al Shaer D, Al Musaimi O, Albericio F, de la Torre BG. 2021 FDA TIDES (Peptides and Oligonucleotides) Harvest. Pharmaceuticals (Basel). 2022;15(2):222.
Article CAS PubMed PubMed Central Google Scholar
Al Musaimi O, Al Shaer D, Albericio F, de la Torre BG. 2022 FDA TIDES (Peptides and Oligonucleotides) Harvest. Pharmaceuticals. 2023;16(3):336.
Article PubMed PubMed Central Google Scholar
Dhuri K, Bechtold C, Quijano E, Pham H, Gupta A, Vikram A. Antisense oligonucleotides: an emerging area in drug discovery and development. J Clin Med. 2020;9(6):2004.
Article CAS PubMed PubMed Central Google Scholar
Rossi JJ, Rossi DJ. siRNA Drugs: Here to Stay. Mol Ther. 2021;29(2):431–2.
Article CAS PubMed PubMed Central Google Scholar
Ni S, Zhuo Z, Pan Y, Yu Y, Li F, Liu J. Recent progress in aptamer discoveries and modifications for therapeutic applications. ACS Appl Mater Interfaces. 2021;13(8):9500–19.
Article CAS PubMed Google Scholar
Pardi N, Hogan MJ, Porter FW, Weissman D. mRNA vaccines — a new era in vaccinology. Nat Rev Drug Discov. 2018;17(4):261–79.
Article CAS PubMed PubMed Central Google Scholar
Park JW, Lagniton PNP, Liu Y, Xu RH. mRNA vaccines for COVID-19: what, why and how. Int J Biol Sci. 2021;17(6):1446–60.
Article CAS PubMed PubMed Central Google Scholar
Hong M, Clubb JD, Chen YY. Engineering CAR-T cells for next-generation cancer therapy. Cancer Cell. 2020;38(4):473–88.
Article CAS PubMed Google Scholar
Behr M, Zhou J, Xu B, Zhang H. In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges. Acta Pharmaceutica Sinica B. 2021;11(8):2150–71.
Article CAS PubMed PubMed Central Google Scholar
Fellmann C, Gowen BG, Lin PC, Doudna JA, Corn JE. Cornerstones of CRISPR-Cas in drug development and therapy. Nat Rev Drug Discov. 2017;16(2):89–100.
Article CAS PubMed Google Scholar
Philippidis A. CASGEVY Makes history as FDA approves first CRISPR/Cas9 genome edited therapy. Human Gene Ther. 2024;35(1–2):1–4.
Ivleva VB, Yu YQ, Gilar M. Ultra-performance liquid chromatography/tandem mass spectrometry (UPLC/MS/MS) and UPLC/MSE analysis of RNA oligonucleotides. Rapid Commun Mass Spectr. 2010;24(17):2631–40.
Wei B, Goyon A, Zhang K. Analysis of therapeutic nucleic acids by capillary electrophoresis. J Pharm Biomed Anal. 2022;219:114928.
Article CAS PubMed Google Scholar
Gallagher SR, Desjardins PR. Quantitation of DNA and RNA with absorption and fluorescence spectroscopy. Curr Protoc Mol Biol. 2006;76(1):A.3D.1-A.3D.21.
Orlando C, Pinzani P, Pazzagli M. Developments in quantitative PCR. 1998;36(5):255–69.
Wong ML, Medrano JF. Real-time PCR for mRNA quantitation. BioTechniques. 2005;39(1):75–85.
Article CAS PubMed Google Scholar
Foley KP, Leonard MW, Engel JD. Quantitation of RNA using the polymerase chain reaction. Trends Gen. 1993;9(11):380–5.
Wei X, Dai G, Marcucci G, Liu Z, Hoyt D, Blum W. A specific picomolar hybridization-based ELISA assay for the determination of phosphorothioate oligonucleotides in plasma and cellular matrices. Pharm Res. 2006;23(6):1251–64.
Article CAS PubMed Google Scholar
Yu RZ, Baker B, Chappell A, Geary RS, Cheung E, Levin AA. Development of an ultrasensitive noncompetitive hybridization-ligation enzyme-linked immunosorbent assay for the determination of phosphorothioate oligodeoxynucleotide in plasma. Anal Biochem. 2002;304(1):19–25.
Article CAS PubMed Google Scholar
Buermans HPJ, den Dunnen JT. Next generation sequencing technology: Advances and applications. Biochimica et Biophysica Acta BBA Molecular Basis of Disease. 2014;1842(10):1932–41.
Article CAS PubMed Google Scholar
Alfonzo JD, Brown JA, Byers PH, Cheung VG, Maraia RJ, Ross RL. A call for direct sequencing of full-length RNAs to identify all modifications. Nat Genet. 2021;53(8):1113–6.
Article CAS PubMed Google Scholar
Dard-Dascot C, Naquin D, d’Aubenton-Carafa Y, Alix K, Thermes C, van Dijk E. Systematic comparison of small RNA library preparation protocols for next-generation sequencing. BMC Genomics. 2018;19(1):118.
Article PubMed PubMed Central Google Scholar
Abdullah AM, Sommers C, Hawes J, Rodriguez JD, Yang K. Tandem mass spectrometric sequence characterization of synthetic thymidine-rich oligonucleotides. J Mass Spectr. 2022;57(4):e4819.
Bahr U, Aygün H, Karas M. Sequencing of single and double stranded RNA oligonucleotides by acid hydrolysis and MALDI mass spectrometry. Anal Chem. 2009;81(8):3173–9.
Article CAS PubMed Google Scholar
Novikova D, Sagaidak A, Vorona S, Tribulovich V. A visual compendium of principal modifications within the nucleic acid sugar phosphate backbone. Molecules. 2024;29(13):3025.
Article CAS PubMed PubMed Central Google Scholar
Goyon A, Scott B, Kurita K, Crittenden CM, Shaw D, Lin A. Full sequencing of CRISPR/Cas9 Single Guide RNA (sgRNA) via parallel ribonuclease digestions and hydrophilic interaction liquid chromatography–high-resolution mass spectrometry analysis. Anal Chem. 2021;93(44):14792–801.
Article CAS PubMed Google Scholar
Goyon A, Scott B, Kurita K, Maschinot C, Meyer K, Yehl P. On-line sequencing of CRISPR Guide RNAs and their impurities via the use of immobilized ribonuclease cartridges attached to a 2D/3D-LC–MS system. Anal Chem. 2022;94(2):1169–77.
Article CAS PubMed Google Scholar
Chin S, Goyon A, Zhang K, Kurita KL. Middle-out sequence confirmation of CRISPR/Cas9 single guide RNA (sgRNA) using DNA primers and ribonuclease T1 digestion. Anal Bioanal Chem. 2023;415(14):2809–18.
Article CAS PubMed Google Scholar
Vanhinsbergh CJ, Criscuolo A, Sutton JN, Murphy K, Williamson AJK, Cook K. Characterization and sequence mapping of large RNA and mRNA therapeutics using mass spectrometry. Anal Chem. 2022;94(20):7339–49.
Article CAS PubMed PubMed Central Google Scholar
Kenderdine T, McIntyre W, Yassaghi G, Rollo D, Bunkowski A, Goerlach L. Integrating internal fragments in the interpretation of top-down sequencing data of larger oligonucleotides. J Am Soc Mass Spectrom. 2023;34(10):2296–307.
Article CAS PubMed Google Scholar
Crittenden CM, Lanzillotti MB, Chen B. Top-down mass spectrometry of synthetic single guide ribonucleic acids enabled by facile sample clean-up. Anal Chem. 2023;95(6):3180–6.
留言 (0)