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For: Fagiuoli S, Daina E, D'Antiga L, Colledan M, Remuzzi G. Monogenic diseases that can be cured by liver transplantation. J Hepatol. 2013;59:595-612. [PMID: 23578885 DOI: 10.1016/j.jhep.2013.04.004] [Cited by in Crossref: 81] [Cited by in F6Publishing: 65] [Article Influence: 9.0] [Reference Citation Analysis]
Number Citing Articles
1 Gough A, Soto-Gutierrez A, Vernetti L, Ebrahimkhani MR, Stern AM, Taylor DL. Human biomimetic liver microphysiology systems in drug development and precision medicine. Nat Rev Gastroenterol Hepatol 2021;18:252-68. [PMID: 33335282 DOI: 10.1038/s41575-020-00386-1] [Cited by in Crossref: 5] [Cited by in F6Publishing: 3] [Article Influence: 2.5] [Reference Citation Analysis]
2 Ronzitti G, Bortolussi G, van Dijk R, Collaud F, Charles S, Leborgne C, Vidal P, Martin S, Gjata B, Sola MS, van Wittenberghe L, Vignaud A, Veron P, Bosma PJ, Muro AF, Mingozzi F. A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome. Mol Ther Methods Clin Dev 2016;3:16049. [PMID: 27722180 DOI: 10.1038/mtm.2016.49] [Cited by in Crossref: 32] [Cited by in F6Publishing: 30] [Article Influence: 5.3] [Reference Citation Analysis]
3 Bortolussi G, Baj G, Vodret S, Viviani G, Bittolo T, Muro AF. Age-dependent pattern of cerebellar susceptibility to bilirubin neurotoxicity in vivo in mice. Dis Model Mech 2014;7:1057-68. [PMID: 25062689 DOI: 10.1242/dmm.016535] [Cited by in Crossref: 28] [Cited by in F6Publishing: 24] [Article Influence: 3.5] [Reference Citation Analysis]
4 European Association for the Study of the Liver. EASL Clinical Practice Guidelines: Liver transplantation. J Hepatol. 2016;64:433-485. [PMID: 26597456 DOI: 10.1016/j.jhep.2015.10.006] [Cited by in Crossref: 390] [Cited by in F6Publishing: 292] [Article Influence: 55.7] [Reference Citation Analysis]
5 Najimi M, Defresne F, Sokal EM. Concise Review: Updated Advances and Current Challenges in Cell Therapy for Inborn Liver Metabolic Defects. Stem Cells Transl Med 2016;5:1117-25. [PMID: 27245366 DOI: 10.5966/sctm.2015-0260] [Cited by in Crossref: 22] [Cited by in F6Publishing: 20] [Article Influence: 3.7] [Reference Citation Analysis]
6 Celik N, Squires JE, Soltys K, Vockley J, Shellmer DA, Chang W, Strauss K, McKiernan P, Ganoza A, Sindhi R, Bond G, Mazariegos G, Khanna A. Domino liver transplantation for select metabolic disorders: Expanding the living donor pool. JIMD Rep. 2019;48:83-89. [PMID: 31392117 DOI: 10.1002/jmd2.12053] [Cited by in Crossref: 5] [Cited by in F6Publishing: 6] [Article Influence: 1.7] [Reference Citation Analysis]
7 Yovchev M, Jaber FL, Lu Z, Patel S, Locker J, Rogler LE, Murray JW, Sudol M, Dabeva MD, Zhu L, Shafritz DA. Experimental Model for Successful Liver Cell Therapy by Lenti TTR-YapERT2 Transduced Hepatocytes with Tamoxifen Control of Yap Subcellular Location. Sci Rep 2016;6:19275. [PMID: 26763940 DOI: 10.1038/srep19275] [Cited by in Crossref: 7] [Cited by in F6Publishing: 5] [Article Influence: 1.2] [Reference Citation Analysis]
8 Hammond N, Munkacsi AB, Sturley SL. The complexity of a monogenic neurodegenerative disease: More than two decades of therapeutic driven research into Niemann-Pick type C disease. Biochim Biophys Acta Mol Cell Biol Lipids 2019;1864:1109-23. [PMID: 31002946 DOI: 10.1016/j.bbalip.2019.04.002] [Cited by in Crossref: 24] [Cited by in F6Publishing: 20] [Article Influence: 8.0] [Reference Citation Analysis]
9 Elfar W, Järvinen E, Ji W, Mosorin J, Sega AG, Iuga AC, Lobritto SJ, Konstantino M, Chan A, Finel M, Lakhani SA. A Novel Pathogenic UGT1A1 Variant in a Sudanese Child with Type 1 Crigler-Najjar Syndrome. Drug Metab Dispos 2019;47:45-8. [PMID: 30385458 DOI: 10.1124/dmd.118.084368] [Reference Citation Analysis]
10 Tan A, Florman SS, Schiano TD. Genetic, hematological, and immunological disorders transmissible with liver transplantation. Liver Transpl 2017;23:663-78. [PMID: 28240807 DOI: 10.1002/lt.24755] [Cited by in Crossref: 7] [Article Influence: 1.4] [Reference Citation Analysis]
11 Kattenhorn LM, Tipper CH, Stoica L, Geraghty DS, Wright TL, Clark KR, Wadsworth SC. Adeno-Associated Virus Gene Therapy for Liver Disease. Hum Gene Ther 2016;27:947-61. [PMID: 27897038 DOI: 10.1089/hum.2016.160] [Cited by in Crossref: 58] [Cited by in F6Publishing: 57] [Article Influence: 11.6] [Reference Citation Analysis]
12 Maes M, Crespo Yanguas S, Willebrords J, Cogliati B, Vinken M. Connexin and pannexin signaling in gastrointestinal and liver disease. Transl Res. 2015;166:332-343. [PMID: 26051630 DOI: 10.1016/j.trsl.2015.05.005] [Cited by in Crossref: 28] [Cited by in F6Publishing: 28] [Article Influence: 4.0] [Reference Citation Analysis]
13 Pankowicz FP, Barzi M, Legras X, Hubert L, Mi T, Tomolonis JA, Ravishankar M, Sun Q, Yang D, Borowiak M, Sumazin P, Elsea SH, Bissig-Choisat B, Bissig KD. Reprogramming metabolic pathways in vivo with CRISPR/Cas9 genome editing to treat hereditary tyrosinaemia. Nat Commun 2016;7:12642. [PMID: 27572891 DOI: 10.1038/ncomms12642] [Cited by in Crossref: 72] [Cited by in F6Publishing: 63] [Article Influence: 12.0] [Reference Citation Analysis]
14 Bočkor L, Bortolussi G, Iaconcig A, Chiaruttini G, Tiribelli C, Giacca M, Benvenuti F, Zentilin L, Muro AF. Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler-Najjar Syndrome Type I. Gene Ther 2017;24:649-60. [PMID: 28805798 DOI: 10.1038/gt.2017.75] [Cited by in Crossref: 19] [Cited by in F6Publishing: 15] [Article Influence: 3.8] [Reference Citation Analysis]
15 Ruiz de Galarreta M, Lujambio A. Therapeutic editing of hepatocyte genome in vivo. J Hepatol 2017;67:818-28. [PMID: 28527665 DOI: 10.1016/j.jhep.2017.05.012] [Cited by in Crossref: 11] [Cited by in F6Publishing: 9] [Article Influence: 2.2] [Reference Citation Analysis]
16 Chu TH, Chien YH, Lin HY, Liao HC, Ho HJ, Lai CJ, Chiang CC, Lin NC, Yang CF, Hwu WL, Lee NC, Lin SP, Liu CS, Hu RH, Ho MC, Niu DM. Methylmalonic acidemia/propionic acidemia - the biochemical presentation and comparing the outcome between liver transplantation versus non-liver transplantation groups. Orphanet J Rare Dis 2019;14:73. [PMID: 30940196 DOI: 10.1186/s13023-019-1045-1] [Cited by in Crossref: 9] [Cited by in F6Publishing: 9] [Article Influence: 3.0] [Reference Citation Analysis]
17 Wei L, Zhang HM, Wan CD, Qu W, Zeng ZG, Liu Y, Xiong J, Sun LY, Zhu ZJ. Auxiliary Liver Graft Can Be Protected From HBV Infection in HBsAg Positive Blood Circulation. Front Med (Lausanne) 2021;8:726502. [PMID: 34513885 DOI: 10.3389/fmed.2021.726502] [Reference Citation Analysis]
18 Pérez B, Vilageliu L, Grinberg D, Desviat LR. Antisense mediated splicing modulation for inherited metabolic diseases: challenges for delivery. Nucleic Acid Ther 2014;24:48-56. [PMID: 24506780 DOI: 10.1089/nat.2013.0453] [Cited by in Crossref: 10] [Cited by in F6Publishing: 9] [Article Influence: 1.3] [Reference Citation Analysis]
19 Waldvogel-abramovski S, Waeber G, Gassner C, Buser A, Frey BM, Favrat B, Tissot J. Iron and transfusion medicine. Blood Reviews 2013;27:289-95. [DOI: 10.1016/j.blre.2013.10.001] [Cited by in Crossref: 17] [Cited by in F6Publishing: 12] [Article Influence: 1.9] [Reference Citation Analysis]
20 Bortolussi G, Zentillin L, Vaníkova J, Bockor L, Bellarosa C, Mancarella A, Vianello E, Tiribelli C, Giacca M, Vitek L. Life-long correction of hyperbilirubinemia with a neonatal liver-specific AAV-mediated gene transfer in a lethal mouse model of Crigler-Najjar Syndrome. Hum Gene Ther. 2014;25:844-855. [PMID: 25072305 DOI: 10.1089/hum.2013.233] [Cited by in Crossref: 50] [Cited by in F6Publishing: 44] [Article Influence: 7.1] [Reference Citation Analysis]
21 D'Antiga L, Colledan M. Surgical gene therapy by domino auxiliary liver transplantation. Liver Transpl 2015;21:1338-9. [PMID: 26335346 DOI: 10.1002/lt.24326] [Cited by in Crossref: 8] [Cited by in F6Publishing: 4] [Article Influence: 1.3] [Reference Citation Analysis]
22 Gu P, Yang Q, Chen B, Bie YN, Liu W, Tian Y, Luo H, Xu T, Liang C, Ye X, Liu Y, Tang X, Gu W. Genetically blocking HPD via CRISPR-Cas9 protects against lethal liver injury in a pig model of tyrosinemia type I. Mol Ther Methods Clin Dev 2021;21:530-47. [PMID: 33997102 DOI: 10.1016/j.omtm.2021.04.002] [Reference Citation Analysis]
23 Meliani A, Boisgerault F, Hardet R, Marmier S, Collaud F, Ronzitti G, Leborgne C, Costa Verdera H, Simon Sola M, Charles S, Vignaud A, van Wittenberghe L, Manni G, Christophe O, Fallarino F, Roy C, Michaud A, Ilyinskii P, Kishimoto TK, Mingozzi F. Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration. Nat Commun 2018;9:4098. [PMID: 30291246 DOI: 10.1038/s41467-018-06621-3] [Cited by in Crossref: 87] [Cited by in F6Publishing: 85] [Article Influence: 21.8] [Reference Citation Analysis]
24 Guo X, Chung T, Qin Y, Zheng J, Zheng H, Sheng L, Wynn T, Chang L. Hemophilia Gene Therapy: New Development from Bench to Bed Side. CGT 2019;19:264-73. [DOI: 10.2174/1566523219666190924121836] [Cited by in Crossref: 6] [Cited by in F6Publishing: 3] [Article Influence: 2.0] [Reference Citation Analysis]
25 Collaud F, Bortolussi G, Guianvarc'h L, Aronson SJ, Bordet T, Veron P, Charles S, Vidal P, Sola MS, Rundwasser S, Dufour DG, Lacoste F, Luc C, Wittenberghe LV, Martin S, Le Bec C, Bosma PJ, Muro AF, Ronzitti G, Hebben M, Mingozzi F. Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome. Mol Ther Methods Clin Dev 2019;12:157-74. [PMID: 30705921 DOI: 10.1016/j.omtm.2018.12.011] [Cited by in Crossref: 17] [Cited by in F6Publishing: 19] [Article Influence: 4.3] [Reference Citation Analysis]
26 Schielke A, Conti F, Goumard C, Perdigao F, Calmus Y, Scatton O. Liver transplantation using grafts with rare metabolic disorders. Dig Liver Dis. 2015;47:261-270. [PMID: 25498135 DOI: 10.1016/j.dld.2014.11.004] [Cited by in Crossref: 17] [Cited by in F6Publishing: 13] [Article Influence: 2.1] [Reference Citation Analysis]
27 Nami F, Basiri M, Satarian L, Curtiss C, Baharvand H, Verfaillie C. Strategies for In Vivo Genome Editing in Nondividing Cells. Trends Biotechnol 2018;36:770-86. [PMID: 29685818 DOI: 10.1016/j.tibtech.2018.03.004] [Cited by in Crossref: 29] [Cited by in F6Publishing: 28] [Article Influence: 7.3] [Reference Citation Analysis]
28 Govil S, Shanmugam NP, Reddy MS, Narasimhan G, Rela M. A metabolic chimera: Two defective genotypes make a normal phenotype. Liver Transpl. 2015;21:1453-1454. [PMID: 26122900 DOI: 10.1002/lt.24202] [Cited by in Crossref: 18] [Cited by in F6Publishing: 13] [Article Influence: 2.6] [Reference Citation Analysis]
29 Zhu X, Zhang B, He Y, Bao J. Liver Organoids: Formation Strategies and Biomedical Applications. Tissue Eng Regen Med 2021;18:573-85. [PMID: 34132985 DOI: 10.1007/s13770-021-00357-w] [Cited by in Crossref: 1] [Article Influence: 1.0] [Reference Citation Analysis]
30 Sirrs S, Hannah-shmouni F, Nantel S, Neuberger J, Yoshida EM. Transplantation as disease modifying therapy in adults with inherited metabolic disorders. J Inherit Metab Dis 2018;41:885-96. [DOI: 10.1007/s10545-018-0141-z] [Cited by in Crossref: 6] [Cited by in F6Publishing: 6] [Article Influence: 1.5] [Reference Citation Analysis]
31 Habeb AM, Deeb A, Johnson M, Abdullah M, Abdulrasoul M, Al-Awneh H, Al-Maghamsi MS, Al-Murshedi F, Al-Saif R, Al-Sinani S, Ramadan D, Tfayli H, Flanagan SE, Ellard S. Liver disease and other comorbidities in Wolcott-Rallison syndrome: different phenotype and variable associations in a large cohort. Horm Res Paediatr 2015;83:190-7. [PMID: 25659842 DOI: 10.1159/000369804] [Cited by in Crossref: 17] [Cited by in F6Publishing: 13] [Article Influence: 2.4] [Reference Citation Analysis]
32 Park Y, Kim H, Kim B, Xu W, Lee H, Wang H. Auxiliary Partial Orthotopic Liver Transplantation as a Treatment for Hemophilia A: A Case Report. Transplantation Proceedings 2015;47:161-4. [DOI: 10.1016/j.transproceed.2014.08.047] [Cited by in Crossref: 5] [Cited by in F6Publishing: 1] [Article Influence: 0.7] [Reference Citation Analysis]
33 Reddy MS, Rajalingam R, Rela M. Revisiting APOLT for Metabolic Liver Disease: A New Look at an Old Idea. Transplantation 2017;101:260-6. [PMID: 27607532 DOI: 10.1097/TP.0000000000001472] [Cited by in Crossref: 15] [Cited by in F6Publishing: 3] [Article Influence: 3.0] [Reference Citation Analysis]
34 Knotek M, Novak R, Jaklin-Kelez A, Mrzljak A. Combined liver-kidney transplantation for rare diseases. World J Hepatol 2020; 12(10): 722-737 [PMID: 33200012 DOI: 10.4254/wjh.v12.i10.722] [Cited by in Crossref: 1] [Cited by in F6Publishing: 1] [Article Influence: 0.5] [Reference Citation Analysis]
35 Prior N, Inacio P, Huch M. Liver organoids: from basic research to therapeutic applications. Gut 2019;68:2228-37. [PMID: 31300517 DOI: 10.1136/gutjnl-2019-319256] [Cited by in Crossref: 78] [Cited by in F6Publishing: 71] [Article Influence: 26.0] [Reference Citation Analysis]
36 Nuciforo S, Heim MH. Organoids to model liver disease. JHEP Rep 2021;3:100198. [PMID: 33241206 DOI: 10.1016/j.jhepr.2020.100198] [Cited by in Crossref: 9] [Cited by in F6Publishing: 8] [Article Influence: 4.5] [Reference Citation Analysis]
37 Celik N, Kelly B, Soltys K, Squires JE, Vockley J, Shellmer DA, Strauss K, McKiernan P, Ganoza A, Sindhi R, Bond G, Mazariegos G, Khanna A. Technique and outcome of domino liver transplantation from patients with maple syrup urine disease: Expanding the donor pool for live donor liver transplantation. Clin Transplant. 2019;33:e13721. [PMID: 31556146 DOI: 10.1111/ctr.13721] [Cited by in Crossref: 6] [Cited by in F6Publishing: 6] [Article Influence: 2.0] [Reference Citation Analysis]
38 Vaisitti T, Peritore D, Magistroni P, Ricci A, Lombardini L, Gringeri E, Catalano S, Spada M, Sciveres M, Di Giorgio A, Limongelli G, Varrenti M, Gerosa G, Terzi A, Pace Napoleone C, Amodeo A, Ragni L, Strologo LD, Benetti E, Fontana I, Testa S, Peruzzi L, Mitrotti A, Abbate S, Comai G, Gotti E, Schiavon M, Boffini M, De Angelis D, Bertani A, Pinelli D, Torre M, Poggi C, Deaglio S, Cardillo M, Amoroso A; Italian Pediatric Transplant Centers. The frequency of rare and monogenic diseases in pediatric organ transplant recipients in Italy. Orphanet J Rare Dis 2021;16:374. [PMID: 34481500 DOI: 10.1186/s13023-021-02013-x] [Reference Citation Analysis]
39 Zabaleta N, Salas D, Paramo M, Hommel M, Sier-Ferreira V, Hernandez-Alcoceba R, Prieto J, Bilbao JI, Gonzalez-Aseguinolaza G. Improvement of Adeno-Associated Virus-Mediated Liver Transduction Efficacy by Regional Administration in Macaca fascicularis. Hum Gene Ther Clin Dev 2017;28:68-73. [PMID: 28285544 DOI: 10.1089/humc.2016.183] [Cited by in Crossref: 4] [Cited by in F6Publishing: 4] [Article Influence: 0.8] [Reference Citation Analysis]
40 Piccolo P, Brunetti-Pierri N. Gene therapy for inherited diseases of liver metabolism. Hum Gene Ther 2015;26:186-92. [PMID: 25830689 DOI: 10.1089/hum.2015.029] [Cited by in Crossref: 9] [Cited by in F6Publishing: 9] [Article Influence: 1.3] [Reference Citation Analysis]
41 Jiang YZ, Sun LY, Zhu ZJ, Wei L, Qu W, Zeng ZG, Liu Y, Tan YL, He EH, Xu RF, Zhang L, Wang J, Chen XJ. Perioperative characteristics and management of liver transplantation for isolated methylmalonic acidemia-the largest experience in China. Hepatobiliary Surg Nutr 2019;8:470-9. [PMID: 31673536 DOI: 10.21037/hbsn.2019.03.04] [Cited by in Crossref: 5] [Cited by in F6Publishing: 4] [Article Influence: 1.7] [Reference Citation Analysis]
42 Porro F, Bortolussi G, Barzel A, De Caneva A, Iaconcig A, Vodret S, Zentilin L, Kay MA, Muro AF. Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model. EMBO Mol Med 2017;9:1346-55. [PMID: 28751579 DOI: 10.15252/emmm.201707601] [Cited by in Crossref: 25] [Cited by in F6Publishing: 19] [Article Influence: 6.3] [Reference Citation Analysis]
43 Berraondo P, Martini PGV, Avila MA, Fontanellas A. Messenger RNA therapy for rare genetic metabolic diseases. Gut 2019;68:1323-30. [PMID: 30796097 DOI: 10.1136/gutjnl-2019-318269] [Cited by in Crossref: 24] [Cited by in F6Publishing: 24] [Article Influence: 8.0] [Reference Citation Analysis]
44 Nicastro E, D'antiga L. Next generation sequencing in pediatric hepatology and liver transplantation. Liver Transpl 2018;24:282-93. [DOI: 10.1002/lt.24964] [Cited by in Crossref: 34] [Cited by in F6Publishing: 27] [Article Influence: 8.5] [Reference Citation Analysis]
45 Mitchell E, Ranganathan S, Mckiernan P, Squires RH, Strauss K, Soltys K, Mazariegos G, Squires JE. Hepatic Parenchymal Injury in Crigler-Najjar Type I. Journal of Pediatric Gastroenterology & Nutrition 2018;66:588-94. [DOI: 10.1097/mpg.0000000000001843] [Cited by in Crossref: 11] [Cited by in F6Publishing: 2] [Article Influence: 2.8] [Reference Citation Analysis]
46 Bai J, Li L, Liu H, Liu S, Bai L, Song W, Chen Y, Zheng S, Duan Z. UGT1A1-related Bilirubin Encephalopathy/Kernicterus in Adults. J Clin Transl Hepatol 2021;9:180-6. [PMID: 34007799 DOI: 10.14218/JCTH.2020.00108] [Reference Citation Analysis]
47 De Caneva A, Porro F, Bortolussi G, Sola R, Lisjak M, Barzel A, Giacca M, Kay MA, Vlahoviček K, Zentilin L, Muro AF. Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases. JCI Insight 2019;5:128863. [PMID: 31211694 DOI: 10.1172/jci.insight.128863] [Cited by in Crossref: 10] [Cited by in F6Publishing: 9] [Article Influence: 3.3] [Reference Citation Analysis]
48 Menon J, Vij M, Sachan D, Rammohan A, Shanmugam N, Kaliamoorthy I, Rela M. Pediatric metabolic liver diseases: Evolving role of liver transplantation. World J Transplant 2021; 11(6): 161-179 [PMID: 34164292 DOI: 10.5500/wjt.v11.i6.161] [Cited by in CrossRef: 1] [Article Influence: 1.0] [Reference Citation Analysis]
49 Lipiński P, Ciara E, Jurkiewicz D, Pollak A, Wypchło M, Płoski R, Cielecka-Kuszyk J, Socha P, Pawłowska J, Jankowska I. Targeted Next-Generation Sequencing in Diagnostic Approach to Monogenic Cholestatic Liver Disorders-Single-Center Experience. Front Pediatr 2020;8:414. [PMID: 32793533 DOI: 10.3389/fped.2020.00414] [Cited by in Crossref: 5] [Cited by in F6Publishing: 4] [Article Influence: 2.5] [Reference Citation Analysis]
50 Witzigmann D, Kulkarni JA, Leung J, Chen S, Cullis PR, van der Meel R. Lipid nanoparticle technology for therapeutic gene regulation in the liver. Adv Drug Deliv Rev 2020;159:344-63. [PMID: 32622021 DOI: 10.1016/j.addr.2020.06.026] [Cited by in Crossref: 26] [Cited by in F6Publishing: 25] [Article Influence: 13.0] [Reference Citation Analysis]
51 Squires J, Nguyen C. Complexity of pre-emptive liver transplantation in children with primary hyperoxaluria type 1. Pediatr Transplant 2016;20:604-6. [PMID: 27292943 DOI: 10.1111/petr.12740] [Cited by in Crossref: 2] [Article Influence: 0.3] [Reference Citation Analysis]
52 Honda M, Sakamoto S, Sakamoto R, Matsumoto S, Irie T, Uchida K, Shimata K, Kawabata S, Isono K, Hayashida S, Yamamoto H, Endo F, Inomata Y. Antibody-mediated rejection after ABO-incompatible pediatric living donor liver transplantation for propionic acidemia: A case report. Pediatr Transplantation 2016;20:840-5. [DOI: 10.1111/petr.12722] [Cited by in Crossref: 4] [Cited by in F6Publishing: 4] [Article Influence: 0.7] [Reference Citation Analysis]
53 Sosulski ML, Stiles KM, Frenk EZ, Hart FM, Matsumura Y, De BP, Kaminsky SM, Crystal RG. Gene therapy for alpha 1-antitrypsin deficiency with an oxidant-resistant human alpha 1-antitrypsin. JCI Insight 2020;5:135951. [PMID: 32759494 DOI: 10.1172/jci.insight.135951] [Cited by in Crossref: 2] [Cited by in F6Publishing: 2] [Article Influence: 1.0] [Reference Citation Analysis]
54 Famulari ES, Navarro-Tableros V, Herrera Sanchez MB, Bortolussi G, Gai M, Conti L, Silengo L, Tolosano E, Tetta C, Muro AF, Camussi G, Fagoonee S, Altruda F. Human liver stem cells express UGT1A1 and improve phenotype of immunocompromised Crigler Najjar syndrome type I mice. Sci Rep 2020;10:887. [PMID: 31965023 DOI: 10.1038/s41598-020-57820-2] [Cited by in Crossref: 5] [Cited by in F6Publishing: 3] [Article Influence: 2.5] [Reference Citation Analysis]
55 Shagrani M, Burkholder J, Broering D, Abouelhoda M, Faquih T, El-Kalioby M, Subhani SN, Goljan E, Albar R, Monies D, Mazhar N, AlAbdulaziz BS, Abdelrahman KA, Altassan N, Alkuraya FS. Genetic profiling of children with advanced cholestatic liver disease. Clin Genet. 2017;92:52-61. [PMID: 28039895 DOI: 10.1111/cge.12959] [Cited by in Crossref: 19] [Cited by in F6Publishing: 17] [Article Influence: 3.8] [Reference Citation Analysis]
56 Yap S, Vara R, Morais A. Post-transplantation Outcomes in Patients with PA or MMA: A Review of the Literature. Adv Ther. 2020;37:1866-1896. [PMID: 32270363 DOI: 10.1007/s12325-020-01305-1] [Cited by in Crossref: 6] [Cited by in F6Publishing: 5] [Article Influence: 3.0] [Reference Citation Analysis]
57 Shanmugam NP, Valamparampil JJ, Reddy MS, Al Said KJ, Al-Thihli K, Al-Hashmi N, Al-Jishi E, Isa HMA, Jalan AB, Rela M. Auxiliary Partial Orthotopic Liver Transplantation for Monogenic Metabolic Liver Diseases: Single-Centre Experience. JIMD Rep. 2019;45:29-36. [PMID: 30311140 DOI: 10.1007/8904_2018_137] [Cited by in Crossref: 8] [Cited by in F6Publishing: 5] [Article Influence: 2.0] [Reference Citation Analysis]
58 Spada M, Angelico R, Dionisi-Vici C. Maple Syrup Urine Disease and Domino Liver Transplantation: When and How? Liver Transpl 2019;25:827-8. [PMID: 31038782 DOI: 10.1002/lt.25481] [Cited by in Crossref: 3] [Cited by in F6Publishing: 2] [Article Influence: 1.5] [Reference Citation Analysis]
59 Critelli K, McKiernan P, Vockley J, Mazariegos G, Squires RH, Soltys K, Squires JE. Liver Transplantation for Propionic Acidemia and Methylmalonic Acidemia: Perioperative Management and Clinical Outcomes. Liver Transpl. 2018;24:1260-1270. [PMID: 30080956 DOI: 10.1002/lt.25304] [Cited by in Crossref: 29] [Cited by in F6Publishing: 27] [Article Influence: 9.7] [Reference Citation Analysis]
60 Pipe SW. Gene therapy for hemophilia. Pediatr Blood Cancer 2018;65. [PMID: 29077262 DOI: 10.1002/pbc.26865] [Cited by in Crossref: 23] [Cited by in F6Publishing: 18] [Article Influence: 4.6] [Reference Citation Analysis]
61 Clar J, Mutel E, Gri B, Creneguy A, Stefanutti A, Gaillard S, Ferry N, Beuf O, Mithieux G, Nguyen TH, Rajas F. Hepatic lentiviral gene transfer prevents the long-term onset of hepatic tumours of glycogen storage disease type 1a in mice. Human Molecular Genetics 2015;24:2287-96. [DOI: 10.1093/hmg/ddu746] [Cited by in Crossref: 12] [Cited by in F6Publishing: 13] [Article Influence: 1.7] [Reference Citation Analysis]
62 Cheng Q, He SQ, Gao D, Lei B, Long X, Liang HF, Zhu P, Jin JF, Tang B, Tomlinson S, Wu ZY, Chen XP. Early Application of Auxiliary Partial Orthotopic Liver Transplantation in Murine Model of Wilson Disease. Transplantation 2015;99:2317-24. [PMID: 26018347 DOI: 10.1097/TP.0000000000000787] [Cited by in Crossref: 6] [Cited by in F6Publishing: 1] [Article Influence: 1.0] [Reference Citation Analysis]
63 Aronson SJ, Beuers U, Bosma PJ. Progress and challenges in gene therapy for Crigler–Najjar syndrome. Expert Opinion on Orphan Drugs 2015;3:1387-96. [DOI: 10.1517/21678707.2015.1100991] [Reference Citation Analysis]
64 Tuncel AT, Boy N, Morath MA, Hörster F, Mütze U, Kölker S. Organic acidurias in adults: late complications and management. J Inherit Metab Dis 2018;41:765-76. [PMID: 29335813 DOI: 10.1007/s10545-017-0135-2] [Cited by in Crossref: 11] [Cited by in F6Publishing: 10] [Article Influence: 2.8] [Reference Citation Analysis]
65 Chen M, Wu J, Liang N, Tang L, Chen Y, Chen H, Wei W, Wei T, Huang H, Yi X, Qi M. Identification of a novel SBF2 frameshift mutation in charcot-marie-tooth disease type 4B2 using whole-exome sequencing. Genomics Proteomics Bioinformatics 2014;12:221-7. [PMID: 25462154 DOI: 10.1016/j.gpb.2014.09.003] [Cited by in Crossref: 5] [Cited by in F6Publishing: 5] [Article Influence: 0.6] [Reference Citation Analysis]
66 Menon J, Shanmugam N, Valamparampil JJ, Hakeem A, Vij M, Jalan A, Reddy MS, Rela M. Liver Transplantation: A Safe and Definitive Alternative to Lifelong Nitisinone for Tyrosinemia Type 1. Indian J Pediatr 2021. [PMID: 34398413 DOI: 10.1007/s12098-021-03826-1] [Reference Citation Analysis]
67 Dong C, Song Z, Meng X, Sun C, Wang K, Yang Y, Qin H, Han C, Zhang F, Zheng W, Gao W. Successful living donor liver transplantation plus domino-auxiliary partial orthotopic liver transplantation for pediatric patients with metabolic disorders. Pediatr Surg Int 2020;36:1443-50. [PMID: 33040172 DOI: 10.1007/s00383-020-04756-3] [Cited by in Crossref: 1] [Article Influence: 0.5] [Reference Citation Analysis]
68 George LA, Ragni MV, Rasko JEJ, Raffini LJ, Samelson-Jones BJ, Ozelo M, Hazbon M, Runowski AR, Wellman JA, Wachtel K, Chen Y, Anguela XM, Kuranda K, Mingozzi F, High KA. Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B. Mol Ther 2020;28:2073-82. [PMID: 32559433 DOI: 10.1016/j.ymthe.2020.06.001] [Cited by in Crossref: 25] [Cited by in F6Publishing: 23] [Article Influence: 12.5] [Reference Citation Analysis]