[1] Zabaleta N, Torella L, Weber ND, et al. mRNA and gene editing: Late breaking therapies in liver diseases. Hepatology, 2022, 76(3): 869-887. [2] Witzigmann D, Kulkarni JA, Leung J, et al. Lipid nanoparticle technology for therapeutic gene regulation in the liver. Adv Drug Deliv Rev, 2020, 159: 344-363. [3] Sahin U, Karikó K, Türeci Ö. mRNA-based therapeutics--developing a new class of drugs. Nat Rev Drug Discov, 2014, 13(10): 759-80. [4] Paunovska K, Da Silva Sanchez AJ, Sago CD, et al. Nanoparticles Containing Oxidized Cholesterol Deliver mRNA to the Liver Microenvironment at Clinically Relevant Doses. Adv Mater, 2019, 31(14): e1807748. [5] Sieber S, Grossen P, Uhl P, et al. Zebrafish as a predictive screening model to assess macrophage clearance of liposomes in vivo. Nanomedicine, 2019, 17: 82-93. [6] Rohner E, Yang R, Foo KS, et al. Unlocking the promise of mRNA therapeutics. Nat Biotechnol, 2022, 40(11): 1586-1600. [7] An D, Frassetto A, Jacquinet E, et al. Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemia. EBioMedicine, 2019, 45: 519-528. [8] Cao J, Choi M, Guadagnin E, et al. mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease. Nat Commun, 2021, 12(1): 3090. [9] Hui R W, Mak L Y, Seto W K, et al. RNA interference as a novel treatment strategy for chronic hepatitis B infection. Clin Mol Hepatol, 2022, 28(3): 408-424. [10] Yuen MF, Lim YS, Cloutier D, et al. Preliminary 48-week Safety and Efficacy Data of VIR-2218 Alone and in Combination With Pegylated Interferon Alfa in Participants With Chronic HBV Infection. The 73rd Annual Meeting of the AASLD, 2022. [11] Yuen MF, Lim SG, Plesniak R, et al. Efficacy and Safety of Bepirovirsen in Chronic Hepatitis B Infection. N Engl J Med, 2022. [12] Yuen MF, Heo J, Jang JW, et al. Safety, tolerability and antiviral activity of the antisense oligonucleotide bepirovirsen in patients with chronic hepatitis B: a phase 2 randomized controlled trial. Nat Med, 2021, 27(10): 1725-1734. [13] Barzel A, Paulk NK, Shi Y, et al. Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Nature, 2015, 517(7534): 360-4. [14] De Caneva A, Porro F, Bortolussi G, et al. Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases. JCI Insight, 2019, 5(15). [15] Song CQ, Wang D, Jiang T, et al. In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency. Hum Gene Ther, 2018, 29(8): 853-860. [16] Zabaleta N, Barberia M, Martin-Higueras C, et al. CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I. Nat Commun, 2018, 9(1): 5454. [17] Wang J, Chen R, Zhang R, et al. The gRNA-miRNA-gRNA Ternary Cassette Combining CRISPR/Cas9 with RNAi Approach Strongly Inhibits Hepatitis B Virus Replication. Theranostics, 2017, 7(12): 3090-3105. [18] Carreras A, Pane LS, Nitsch R, et al. In vivo genome and base editing of a human PCSK9 knock-in hypercholesterolemic mouse model. BMC Biol, 2019, 17(1): 4. [19] Beam Therapeutics Presents First In Vivo Data Demonstrating Potential of Multiplex Base Editing Approach to Target Disease Drivers of Chronic Hepatitis B Infection. https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-presents-first-vivo-data-demonstrating. [20] Nelson CE, Wu Y, Gemberling MP, et al. Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy. Nat Med, 2019, 25(3): 427-432. [21] Wei T, Cheng Q, Min YL, et al. Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing. Nat Commun, 2020, 11(1): 3232. [22] Chen R, Huang H, Liu H, et al. Friend or Foe? Evidence Indicates Endogenous Exosomes Can Deliver Functional gRNA and Cas9 Protein. Small, 2019, 15(38): e1902686. [23] Wan T, Zhong J, Pan Q, et al. Exosome-mediated delivery of Cas9 ribonucleoprotein complexes for tissue-specific gene therapy of liver diseases. Sci Adv, 2022, 8(37): eabp9435. |