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For: Borel F, Tang Q, Gernoux G, Greer C, Wang Z, Barzel A, Kay MA, Shultz LD, Greiner DL, Flotte TR, Brehm MA, Mueller C. Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency. Mol Ther 2017;25:2477-89. [PMID: 29032169 DOI: 10.1016/j.ymthe.2017.09.020] [Cited by in Crossref: 35] [Cited by in F6Publishing: 31] [Article Influence: 7.0] [Reference Citation Analysis]
Number Citing Articles
1 Haugabook SJ, Ferrer M, Ottinger EA. In vitro and in vivo translational models for rare liver diseases. Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease 2019;1865:1003-18. [DOI: 10.1016/j.bbadis.2018.07.029] [Cited by in Crossref: 5] [Cited by in F6Publishing: 6] [Article Influence: 1.7] [Reference Citation Analysis]
2 Ploss A, Kapoor A. Animal Models of Hepatitis C Virus Infection. Cold Spring Harb Perspect Med 2020;10:a036970. [PMID: 31843875 DOI: 10.1101/cshperspect.a036970] [Cited by in Crossref: 6] [Cited by in F6Publishing: 8] [Article Influence: 3.0] [Reference Citation Analysis]
3 Smith LJ, Wright J, Clark G, Ul-Hasan T, Jin X, Fong A, Chandra M, St Martin T, Rubin H, Knowlton D, Ellsworth JL, Fong Y, Wong KK Jr, Chatterjee S. Stem cell-derived clade F AAVs mediate high-efficiency homologous recombination-based genome editing. Proc Natl Acad Sci U S A 2018;115:E7379-88. [PMID: 30018062 DOI: 10.1073/pnas.1802343115] [Cited by in Crossref: 23] [Cited by in F6Publishing: 18] [Article Influence: 5.8] [Reference Citation Analysis]
4 Parés M, Fornaguera C, Vila-Julià F, Oh S, Fan SHY, Tam YK, Comes N, Vidal F, Martí R, Borrós S, Barquinero J. Preclinical Assessment of a Gene-Editing Approach in a Mouse Model of Mitochondrial Neurogastrointestinal Encephalomyopathy. Hum Gene Ther 2021;32:1210-23. [PMID: 34498979 DOI: 10.1089/hum.2021.152] [Reference Citation Analysis]
5 VanLith CJ, Guthman RM, Nicolas CT, Allen KL, Liu Y, Chilton JA, Tritz ZP, Nyberg SL, Kaiser RA, Lillegard JB, Hickey RD. Ex Vivo Hepatocyte Reprograming Promotes Homology-Directed DNA Repair to Correct Metabolic Disease in Mice After Transplantation. Hepatol Commun 2019;3:558-73. [PMID: 30976745 DOI: 10.1002/hep4.1315] [Cited by in Crossref: 7] [Cited by in F6Publishing: 11] [Article Influence: 2.3] [Reference Citation Analysis]
6 Song CQ, Wang D, Jiang T, O'Connor K, Tang Q, Cai L, Li X, Weng Z, Yin H, Gao G, Mueller C, Flotte TR, Xue W. In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency. Hum Gene Ther 2018;29:853-60. [PMID: 29597895 DOI: 10.1089/hum.2017.225] [Cited by in Crossref: 31] [Cited by in F6Publishing: 30] [Article Influence: 7.8] [Reference Citation Analysis]
7 Berns KI, Srivastava A. Next Generation of Adeno-Associated Virus Vectors for Gene Therapy for Human Liver Diseases. Gastroenterol Clin North Am 2019;48:319-30. [PMID: 31046978 DOI: 10.1016/j.gtc.2019.02.005] [Cited by in Crossref: 10] [Cited by in F6Publishing: 7] [Article Influence: 3.3] [Reference Citation Analysis]
8 Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019;18:358-378. [PMID: 30710128 DOI: 10.1038/s41573-019-0012-9] [Cited by in Crossref: 332] [Cited by in F6Publishing: 337] [Article Influence: 110.7] [Reference Citation Analysis]
9 Borel F, Mueller C. Design of AAV Vectors for Delivery of RNAi. Methods Mol Biol 2019;1950:3-18. [PMID: 30783965 DOI: 10.1007/978-1-4939-9139-6_1] [Cited by in Crossref: 2] [Cited by in F6Publishing: 2] [Article Influence: 0.7] [Reference Citation Analysis]
10 Ginn SL, Amaya AK, Liao SHY, Zhu E, Cunningham SC, Lee M, Hallwirth CV, Logan GJ, Tay SS, Cesare AJ, Pickett HA, Grompe M, Dilworth K, Lisowski L, Alexander IE. Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes. JHEP Rep 2020;2:100065. [PMID: 32039406 DOI: 10.1016/j.jhepr.2019.100065] [Cited by in Crossref: 7] [Cited by in F6Publishing: 6] [Article Influence: 2.3] [Reference Citation Analysis]
11 Yao J, Yu Y, Nyberg SL. Induced Pluripotent Stem Cells for the Treatment of Liver Diseases: Novel Concepts. Cells Tissues Organs 2020;:1-17. [PMID: 32615573 DOI: 10.1159/000508182] [Cited by in Crossref: 4] [Cited by in F6Publishing: 3] [Article Influence: 2.0] [Reference Citation Analysis]
12 Chandler RJ, Venturoni LE, Liao J, Hubbard BT, Schneller JL, Hoffmann V, Gordo S, Zang S, Ko CW, Chau N, Chiang K, Kay MA, Barzel A, Venditti CP. Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia. Hepatology 2021;73:2223-37. [PMID: 32976669 DOI: 10.1002/hep.31570] [Cited by in Crossref: 3] [Cited by in F6Publishing: 7] [Article Influence: 3.0] [Reference Citation Analysis]
13 Zhou GP, Sun LY, Zhu ZJ. The concept of "domino" in liver and hepatocyte transplantation. Therap Adv Gastroenterol 2020;13:1756284820968755. [PMID: 33149765 DOI: 10.1177/1756284820968755] [Reference Citation Analysis]
14 Tang Q, Gernoux G, Cheng Y, Flotte T, Mueller C. Engraftment of Human Hepatocytes in the PiZ-NSG Mouse Model. Methods Mol Biol 2020;2164:75-85. [PMID: 32607885 DOI: 10.1007/978-1-0716-0704-6_9] [Reference Citation Analysis]
15 Ginn SL, Christina S, Alexander IE. Genome editing in the human liver: Progress and translational considerations. Prog Mol Biol Transl Sci 2021;182:257-88. [PMID: 34175044 DOI: 10.1016/bs.pmbts.2021.01.030] [Reference Citation Analysis]
16 Elias A, Kassis H, Elkader SA, Gritsenko N, Nahmad A, Shir H, Younis L, Shannan A, Aihara H, Prag G, Yagil E, Kolot M. HK022 bacteriophage Integrase mediated RMCE as a potential tool for human gene therapy. Nucleic Acids Res 2020;48:12804-16. [PMID: 33270859 DOI: 10.1093/nar/gkaa1140] [Reference Citation Analysis]
17 Borel F, Sun H, Zieger M, Cox A, Cardozo B, Li W, Oliveira G, Davis A, Gruntman A, Flotte TR, Brodsky MH, Hoffman AM, Elmallah MK, Mueller C. Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema. Proc Natl Acad Sci U S A 2018;115:2788-93. [PMID: 29453277 DOI: 10.1073/pnas.1713689115] [Cited by in Crossref: 37] [Cited by in F6Publishing: 34] [Article Influence: 9.3] [Reference Citation Analysis]
18 Agarwal N, Popovic B, Martucci NJ, Fraunhoffer NA, Soto-Gutierrez A. Biofabrication of Autologous Human Hepatocytes for Transplantation: How Do We Get There? Gene Expr 2019;19:89-95. [PMID: 30143060 DOI: 10.3727/105221618X15350366478989] [Cited by in Crossref: 2] [Cited by in F6Publishing: 1] [Article Influence: 0.5] [Reference Citation Analysis]
19 Maestro S, Weber ND, Zabaleta N, Aldabe R, Gonzalez-Aseguinolaza G. Novel vectors and approaches for gene therapy in liver diseases. JHEP Rep 2021;3:100300. [PMID: 34159305 DOI: 10.1016/j.jhepr.2021.100300] [Cited by in F6Publishing: 1] [Reference Citation Analysis]
20 Chandler RJ, Venditti CP. Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future. Hum Gene Ther 2019;30:1236-44. [PMID: 31303064 DOI: 10.1089/hum.2019.113] [Cited by in Crossref: 8] [Cited by in F6Publishing: 7] [Article Influence: 2.7] [Reference Citation Analysis]
21 van Haasteren J, Hyde SC, Gill DR. Lessons learned from lung and liver in-vivo gene therapy: implications for the future. Expert Opin Biol Ther 2018;18:959-72. [PMID: 30067117 DOI: 10.1080/14712598.2018.1506761] [Cited by in Crossref: 19] [Cited by in F6Publishing: 17] [Article Influence: 4.8] [Reference Citation Analysis]
22 Pye A, Khan S, Whitehouse T, Turner AM. Personalizing liver targeted treatments and transplantation for patients with alpha-1 antitrypsin deficiency. Expert Review of Precision Medicine and Drug Development 2021;6:65-78. [DOI: 10.1080/23808993.2021.1862648] [Reference Citation Analysis]
23 Yenilmez B, Wetoska N, Kelly M, Echeverria D, Min K, Lifshitz L, Alterman JF, Hassler MR, Hildebrand S, DiMarzio C, McHugh N, Vangjeli L, Sousa J, Pan M, Han X, Brehm MA, Khvorova A, Czech MP. An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis. Mol Ther 2021:S1525-0016(21)00578-5. [PMID: 34774753 DOI: 10.1016/j.ymthe.2021.11.007] [Reference Citation Analysis]
24 Flotte TR. Alpha-1 Antitrypsin Deficiency as a Candidate for Gene Editing. Hum Gene Ther 2018;29:843-4. [PMID: 30096037 DOI: 10.1089/hum.2018.29070.trf] [Cited by in Crossref: 2] [Article Influence: 0.5] [Reference Citation Analysis]
25 Trevisan M, Masi G, Palù G. Genome editing technologies to treat rare liver diseases. Transl Gastroenterol Hepatol 2020;5:23. [PMID: 32258527 DOI: 10.21037/tgh.2019.10.10] [Cited by in Crossref: 3] [Cited by in F6Publishing: 3] [Article Influence: 1.5] [Reference Citation Analysis]
26 Batista AR, Flotte TR. In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis. Mol Ther 2021;29:2633-4. [PMID: 34433081 DOI: 10.1016/j.ymthe.2021.08.014] [Reference Citation Analysis]
27 Craig TJ, Henao MP. Advances in managing COPD related to α1 -antitrypsin deficiency: An under-recognized genetic disorder. Allergy 2018;73:2110-21. [PMID: 29984428 DOI: 10.1111/all.13558] [Cited by in Crossref: 4] [Cited by in F6Publishing: 6] [Article Influence: 1.0] [Reference Citation Analysis]
28 Bissig KD, Han W, Barzi M, Kovalchuk N, Ding L, Fan X, Pankowicz FP, Zhang QY, Ding X. P450-Humanized and Human Liver Chimeric Mouse Models for Studying Xenobiotic Metabolism and Toxicity. Drug Metab Dispos 2018;46:1734-44. [PMID: 30093418 DOI: 10.1124/dmd.118.083303] [Cited by in Crossref: 23] [Cited by in F6Publishing: 21] [Article Influence: 5.8] [Reference Citation Analysis]
29 Zabaleta N, Hommel M, Salas D, Gonzalez-aseguinolaza G. Genetic-Based Approaches to Inherited Metabolic Liver Diseases. Human Gene Therapy 2019;30:1190-203. [DOI: 10.1089/hum.2019.140] [Cited by in Crossref: 11] [Cited by in F6Publishing: 11] [Article Influence: 3.7] [Reference Citation Analysis]
30 Strings-Ufombah V, Malerba A, Kao SC, Harbaran S, Roth F, Cappellari O, Lu-Nguyen N, Takahashi K, Mukadam S, Kilfoil G, Kloth C, Roelvink P, Dickson G, Trollet C, Suhy D. BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy. Mol Ther Nucleic Acids 2021;24:67-78. [PMID: 33738139 DOI: 10.1016/j.omtn.2021.02.017] [Reference Citation Analysis]
31 Dasgupta I, Flotte TR, Keeler AM. CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing. Hum Gene Ther 2021;32:275-93. [PMID: 33750221 DOI: 10.1089/hum.2021.013] [Cited by in Crossref: 5] [Cited by in F6Publishing: 3] [Article Influence: 5.0] [Reference Citation Analysis]
32 Wang D, Li J, Song CQ, Tran K, Mou H, Wu PH, Tai PWL, Mendonca CA, Ren L, Wang BY, Su Q, Gessler DJ, Zamore PD, Xue W, Gao G. Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice. Nat Biotechnol 2018;36:839-42. [PMID: 30102296 DOI: 10.1038/nbt.4219] [Cited by in Crossref: 20] [Cited by in F6Publishing: 21] [Article Influence: 5.0] [Reference Citation Analysis]
33 VanLith C, Guthman R, Nicolas CT, Allen K, Du Z, Joo DJ, Nyberg SL, Lillegard JB, Hickey RD. Curative Ex Vivo Hepatocyte-Directed Gene Editing in a Mouse Model of Hereditary Tyrosinemia Type 1. Hum Gene Ther 2018;29:1315-26. [PMID: 29764210 DOI: 10.1089/hum.2017.252] [Cited by in Crossref: 20] [Cited by in F6Publishing: 16] [Article Influence: 5.0] [Reference Citation Analysis]
34 Curran M, Mairesse M, Matas-Céspedes A, Bareham B, Pellegrini G, Liaunardy A, Powell E, Sargeant R, Cuomo E, Stebbings R, Betts CJ, Saeb-Parsy K. Recent Advancements and Applications of Human Immune System Mice in Preclinical Immuno-Oncology. Toxicol Pathol 2020;48:302-16. [PMID: 31847725 DOI: 10.1177/0192623319886304] [Cited by in Crossref: 7] [Cited by in F6Publishing: 7] [Article Influence: 2.3] [Reference Citation Analysis]
35 Kaserman JE, Wilson AA. Patient-Derived Induced Pluripotent Stem Cells for Alpha-1 Antitrypsin Deficiency Disease Modeling and Therapeutic Discovery. Chronic Obstr Pulm Dis 2018;5:258-66. [PMID: 30723783 DOI: 10.15326/jcopdf.5.4.2017.0179] [Cited by in Crossref: 2] [Cited by in F6Publishing: 1] [Article Influence: 0.5] [Reference Citation Analysis]