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For: Chuah MK, Petrus I, De Bleser P, Le Guiner C, Gernoux G, Adjali O, Nair N, Willems J, Evens H, Rincon MY, Matrai J, Di Matteo M, Samara-Kuko E, Yan B, Acosta-Sanchez A, Meliani A, Cherel G, Blouin V, Christophe O, Moullier P, Mingozzi F, VandenDriessche T. Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates. Mol Ther 2014;22:1605-13. [PMID: 24954473 DOI: 10.1038/mt.2014.114] [Cited by in Crossref: 51] [Cited by in F6Publishing: 55] [Article Influence: 6.4] [Reference Citation Analysis]
Number Citing Articles
1 Castaman G, Di Minno G, De Cristofaro R, Peyvandi F. The Arrival of Gene Therapy for Patients with Hemophilia A. IJMS 2022;23:10228. [DOI: 10.3390/ijms231810228] [Reference Citation Analysis]
2 Jäschke N, Büning H. Adeno-Associated Virus Vector Design-Moving the Adeno-Associated Virus to a Bioengineered Therapeutic Nanoparticle. Hematol Oncol Clin North Am 2022;36:667-85. [PMID: 35778330 DOI: 10.1016/j.hoc.2022.04.002] [Reference Citation Analysis]
3 Marino M, Holt MG. AAV Vector-Mediated Antibody Delivery (A-MAD) in the Central Nervous System. Front Neurol 2022;13:870799. [DOI: 10.3389/fneur.2022.870799] [Reference Citation Analysis]
4 Sung Y, Wang T, Lin T, Lin S. Optogenetics in cardiology: methodology and future applications. Int J Arrhythm 2022;23. [DOI: 10.1186/s42444-022-00060-4] [Reference Citation Analysis]
5 Zhang X, Chai Z, Lee Dobbins A, Itano MS, Askew C, Miao Z, Niu H, Samulski RJ, Li C. Customized blood-brain barrier shuttle peptide to increase AAV9 vector crossing the BBB and augment transduction in the brain. Biomaterials 2021;281:121340. [PMID: 34998171 DOI: 10.1016/j.biomaterials.2021.121340] [Cited by in Crossref: 2] [Cited by in F6Publishing: 2] [Article Influence: 2.0] [Reference Citation Analysis]
6 Mnyandu N, Limani SW, Arbuthnot P, Maepa MB. Advances in designing Adeno-associated viral vectors for development of anti-HBV gene therapeutics. Virol J 2021;18:247. [PMID: 34903258 DOI: 10.1186/s12985-021-01715-9] [Cited by in F6Publishing: 1] [Reference Citation Analysis]
7 Brown D, Altermatt M, Dobreva T, Chen S, Wang A, Thomson M, Gradinaru V. Deep Parallel Characterization of AAV Tropism and AAV-Mediated Transcriptional Changes via Single-Cell RNA Sequencing. Front Immunol 2021;12:730825. [PMID: 34759919 DOI: 10.3389/fimmu.2021.730825] [Cited by in F6Publishing: 6] [Reference Citation Analysis]
8 Chis AA, Dobrea CM, Rus LL, Frum A, Morgovan C, Butuca A, Totan M, Juncan AM, Gligor FG, Arseniu AM. Dendrimers as Non-Viral Vectors in Gene-Directed Enzyme Prodrug Therapy. Molecules 2021;26:5976. [PMID: 34641519 DOI: 10.3390/molecules26195976] [Cited by in F6Publishing: 3] [Reference Citation Analysis]
9 Baik AD, Calafati P, Zhang X, Aaron NA, Mehra A, Moller-Tank S, Miloscio L, Praggastis M, Giovannone N, Pan C, Tang Y, Bridges S, Mujica A, Barbounis P, Yanolatos J, Gale N, Li N, Kyratsous CA, Schoenherr CJ, Murphy AJ, Economides AN, Cygnar KD. Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies. Mol Ther 2021:S1525-0016(21)00414-7. [PMID: 34400331 DOI: 10.1016/j.ymthe.2021.08.020] [Cited by in F6Publishing: 4] [Reference Citation Analysis]
10 Famà R, Borroni E, Merlin S, Airoldi C, Pignani S, Cucci A, Corà D, Bruscaggin V, Scardellato S, Faletti S, Pelicci G, Pinotti M, Walker GE, Follenzi A. Deciphering the Ets-1/2-mediated transcriptional regulation of F8 gene identifies a minimal F8 promoter for hemophilia A gene therapy. Haematologica 2021;106:1624-35. [PMID: 32467137 DOI: 10.3324/haematol.2019.239202] [Reference Citation Analysis]
11 Wagner HJ, Weber W, Fussenegger M. Synthetic Biology: Emerging Concepts to Design and Advance Adeno-Associated Viral Vectors for Gene Therapy. Adv Sci (Weinh) 2021;8:2004018. [PMID: 33977059 DOI: 10.1002/advs.202004018] [Cited by in Crossref: 11] [Cited by in F6Publishing: 9] [Article Influence: 11.0] [Reference Citation Analysis]
12 Nair N, De Wolf D, Nguyen PA, Pham QH, Samara-Kuko E, Landau J, Blouse GE, Chuah MK, VandenDriessche T. Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua. Blood 2021;137:2902-6. [PMID: 33735915 DOI: 10.1182/blood.2020006005] [Cited by in Crossref: 2] [Cited by in F6Publishing: 3] [Article Influence: 2.0] [Reference Citation Analysis]
13 Rambhai HK, Ashby FJ 3rd, Qing K, Srivastava A. Role of Essential Metal Ions in AAV Vector-Mediated Transduction. Mol Ther Methods Clin Dev 2020;18:159-66. [PMID: 32637447 DOI: 10.1016/j.omtm.2020.05.019] [Cited by in Crossref: 1] [Cited by in F6Publishing: 2] [Article Influence: 0.5] [Reference Citation Analysis]
14 Brown HC, Doering CB, Herzog RW, Ling C, Markusic DM, Spencer HT, Srivastava A, Srivastava A. Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B. Hum Gene Ther 2020;31:1114-23. [PMID: 32657150 DOI: 10.1089/hum.2020.099] [Cited by in Crossref: 6] [Cited by in F6Publishing: 10] [Article Influence: 3.0] [Reference Citation Analysis]
15 Ahmed SS, Rubin H, Wang M, Faulkner D, Sengooba A, Dollive SN, Avila N, Ellsworth JL, Lamppu D, Lobikin M, Lotterhand J, Adamson-Small L, Wright T, Seymour A, Francone OL. Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH. Mol Ther Methods Clin Dev 2020;17:568-80. [PMID: 32258219 DOI: 10.1016/j.omtm.2020.03.009] [Cited by in Crossref: 2] [Cited by in F6Publishing: 7] [Article Influence: 1.0] [Reference Citation Analysis]
16 Li C, Samulski RJ. Engineering adeno-associated virus vectors for gene therapy. Nat Rev Genet 2020;21:255-72. [DOI: 10.1038/s41576-019-0205-4] [Cited by in Crossref: 171] [Cited by in F6Publishing: 260] [Article Influence: 85.5] [Reference Citation Analysis]
17 Tipanee J, Di Matteo M, Tulalamba W, Samara-Kuko E, Keirsse J, Van Ginderachter JA, Chuah MK, VandenDriessche T. Validation of miR-20a as a Tumor Suppressor Gene in Liver Carcinoma Using Hepatocyte-Specific Hyperactive piggyBac Transposons. Mol Ther Nucleic Acids 2020;19:1309-29. [PMID: 32160703 DOI: 10.1016/j.omtn.2020.01.015] [Cited by in Crossref: 4] [Cited by in F6Publishing: 6] [Article Influence: 2.0] [Reference Citation Analysis]
18 Richards DY, Winn SR, Dudley S, Nygaard S, Mighell TL, Grompe M, Harding CO. AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria. Mol Ther Methods Clin Dev 2020;17:234-45. [PMID: 31970201 DOI: 10.1016/j.omtm.2019.12.004] [Cited by in Crossref: 15] [Cited by in F6Publishing: 24] [Article Influence: 5.0] [Reference Citation Analysis]
19 Domenger C, Grimm D. Next-generation AAV vectors—do not judge a virus (only) by its cover. Human Molecular Genetics 2019;28:R3-R14. [DOI: 10.1093/hmg/ddz148] [Cited by in Crossref: 38] [Cited by in F6Publishing: 49] [Article Influence: 12.7] [Reference Citation Analysis]
20 Sarcar S, Tulalamba W, Rincon MY, Tipanee J, Pham HQ, Evens H, Boon D, Samara-Kuko E, Keyaerts M, Loperfido M, Berardi E, Jarmin S, In't Veld P, Dickson G, Lahoutte T, Sampaolesi M, De Bleser P, VandenDriessche T, Chuah MK. Next-generation muscle-directed gene therapy by in silico vector design. Nat Commun 2019;10:492. [PMID: 30700722 DOI: 10.1038/s41467-018-08283-7] [Cited by in Crossref: 16] [Cited by in F6Publishing: 22] [Article Influence: 5.3] [Reference Citation Analysis]
21 Leggiero E, Labruna G, Iaffaldano L, Lombardo B, Greco A, Fiorenza D, Gramanzini M, Montanaro D, Baldi A, Cerullo V, Sacchetti L, Pastore L. Helper-dependent adenovirus-mediated gene transfer of a secreted LDL receptor/transferrin chimeric protein reduces aortic atherosclerosis in LDL receptor-deficient mice. Gene Ther 2019;26:121-30. [PMID: 30700805 DOI: 10.1038/s41434-019-0061-z] [Cited by in Crossref: 4] [Cited by in F6Publishing: 5] [Article Influence: 1.3] [Reference Citation Analysis]
22 Merlin S, Follenzi A. Transcriptional Targeting and MicroRNA Regulation of Lentiviral Vectors. Mol Ther Methods Clin Dev 2019;12:223-32. [PMID: 30775404 DOI: 10.1016/j.omtm.2018.12.013] [Cited by in Crossref: 6] [Cited by in F6Publishing: 10] [Article Influence: 2.0] [Reference Citation Analysis]
23 Perrin GQ, Herzog RW, Markusic DM. Update on clinical gene therapy for hemophilia. Blood 2019;133:407-14. [PMID: 30559260 DOI: 10.1182/blood-2018-07-820720] [Cited by in Crossref: 62] [Cited by in F6Publishing: 81] [Article Influence: 15.5] [Reference Citation Analysis]
24 Greig JA, Nordin JML, White JW, Wang Q, Bote E, Goode T, Calcedo R, Wadsworth S, Wang L, Wilson JM. Optimized Adeno-Associated Viral-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques. Hum Gene Ther 2018. [PMID: 29890905 DOI: 10.1089/hum.2018.080] [Cited by in Crossref: 7] [Cited by in F6Publishing: 5] [Article Influence: 1.8] [Reference Citation Analysis]
25 Evens H, Chuah MK, Vandendriessche T. Haemophilia gene therapy: From trailblazer to gamechanger. Haemophilia 2018;24:50-9. [DOI: 10.1111/hae.13494] [Cited by in Crossref: 11] [Cited by in F6Publishing: 11] [Article Influence: 2.8] [Reference Citation Analysis]
26 Herrmann AK, Grimm D. High-Throughput Dissection of AAV-Host Interactions: The Fast and the Curious. J Mol Biol 2018;430:2626-40. [PMID: 29782834 DOI: 10.1016/j.jmb.2018.05.022] [Cited by in Crossref: 13] [Cited by in F6Publishing: 15] [Article Influence: 3.3] [Reference Citation Analysis]
27 Pirih N, Kunej T. An Updated Taxonomy and a Graphical Summary Tool for Optimal Classification and Comprehension of Omics Research. OMICS: A Journal of Integrative Biology 2018;22:337-53. [DOI: 10.1089/omi.2017.0186] [Cited by in Crossref: 6] [Cited by in F6Publishing: 8] [Article Influence: 1.5] [Reference Citation Analysis]
28 Rincon MY, de Vin F, Duqué SI, Fripont S, Castaldo SA, Bouhuijzen-Wenger J, Holt MG. Widespread transduction of astrocytes and neurons in the mouse central nervous system after systemic delivery of a self-complementary AAV-PHP.B vector. Gene Ther 2018;25:83-92. [PMID: 29523880 DOI: 10.1038/s41434-018-0005-z] [Cited by in Crossref: 23] [Cited by in F6Publishing: 27] [Article Influence: 5.8] [Reference Citation Analysis]
29 Singh K, Evens H, Nair N, Rincón MY, Sarcar S, Samara-Kuko E, Chuah MK, VandenDriessche T. Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9. Mol Ther 2018;26:1241-54. [PMID: 29599079 DOI: 10.1016/j.ymthe.2018.02.023] [Cited by in Crossref: 29] [Cited by in F6Publishing: 30] [Article Influence: 7.3] [Reference Citation Analysis]
30 Vidal P, Pagliarani S, Colella P, Costa Verdera H, Jauze L, Gjorgjieva M, Puzzo F, Marmier S, Collaud F, Simon Sola M, Charles S, Lucchiari S, van Wittenberghe L, Vignaud A, Gjata B, Richard I, Laforet P, Malfatti E, Mithieux G, Rajas F, Comi GP, Ronzitti G, Mingozzi F. Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression. Mol Ther 2018;26:890-901. [PMID: 29396266 DOI: 10.1016/j.ymthe.2017.12.019] [Cited by in Crossref: 18] [Cited by in F6Publishing: 17] [Article Influence: 3.6] [Reference Citation Analysis]
31 Vandendriessche T, Chuah MK. Hemophilia Gene Therapy: Ready for Prime Time? Human Gene Therapy 2017;28:1013-23. [DOI: 10.1089/hum.2017.116] [Cited by in Crossref: 20] [Cited by in F6Publishing: 19] [Article Influence: 4.0] [Reference Citation Analysis]
32 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: 18] [Article Influence: 3.8] [Reference Citation Analysis]
33 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: 69] [Article Influence: 11.6] [Reference Citation Analysis]
34 Weinmann J, Grimm D. Next-generation AAV vectors for clinical use: an ever-accelerating race. Virus Genes 2017;53:707-13. [PMID: 28762205 DOI: 10.1007/s11262-017-1502-7] [Cited by in Crossref: 32] [Cited by in F6Publishing: 29] [Article Influence: 6.4] [Reference Citation Analysis]
35 Germain DP. Pseudoxanthoma elasticum. Orphanet J Rare Dis 2017;12:85. [PMID: 28486967 DOI: 10.1186/s13023-017-0639-8] [Cited by in Crossref: 59] [Cited by in F6Publishing: 65] [Article Influence: 11.8] [Reference Citation Analysis]
36 Greig JA, Wang Q, Reicherter AL, Chen SJ, Hanlon AL, Tipper CH, Clark KR, Wadsworth S, Wang L, Wilson JM. Characterization of Adeno-Associated Viral Vector-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice. Hum Gene Ther 2017;28:392-402. [PMID: 28056565 DOI: 10.1089/hum.2016.128] [Cited by in Crossref: 16] [Cited by in F6Publishing: 17] [Article Influence: 3.2] [Reference Citation Analysis]
37 Levy C, Fusil F, Amirache F, Costa C, Girard‐gagnepain A, Negre D, Bernadin O, Garaulet G, Rodriguez A, Nair N, Vandendriessche T, Chuah M, Cosset F, Verhoeyen E. Baboon envelope pseudotyped lentiviral vectors efficiently transduce human B cells and allow active factor IX B cell secretion in vivo in NOD / SCID γc ‐/‐ mice. J Thromb Haemost 2016;14:2478-92. [DOI: 10.1111/jth.13520] [Cited by in Crossref: 25] [Cited by in F6Publishing: 25] [Article Influence: 4.2] [Reference Citation Analysis]
38 Hickmott JW, Chen CY, Arenillas DJ, Korecki AJ, Lam SL, Molday LL, Bonaguro RJ, Zhou M, Chou AY, Mathelier A, Boye SL, Hauswirth WW, Molday RS, Wasserman WW, Simpson EM. PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina. Mol Ther Methods Clin Dev 2016;3:16051. [PMID: 27556059 DOI: 10.1038/mtm.2016.51] [Cited by in Crossref: 8] [Cited by in F6Publishing: 9] [Article Influence: 1.3] [Reference Citation Analysis]
39 El-Shamayleh Y, Ni AM, Horwitz GD. Strategies for targeting primate neural circuits with viral vectors. J Neurophysiol 2016;116:122-34. [PMID: 27052579 DOI: 10.1152/jn.00087.2016] [Cited by in Crossref: 24] [Cited by in F6Publishing: 20] [Article Influence: 4.0] [Reference Citation Analysis]
40 Karjoo Z, Chen X, Hatefi A. Progress and problems with the use of suicide genes for targeted cancer therapy. Adv Drug Deliv Rev 2016;99:113-28. [PMID: 26004498 DOI: 10.1016/j.addr.2015.05.009] [Cited by in Crossref: 98] [Cited by in F6Publishing: 89] [Article Influence: 16.3] [Reference Citation Analysis]
41 Aravalli RN, Steer CJ. Gene editing technology as an approach to the treatment of liver diseases. Expert Opinion on Biological Therapy 2016;16:595-608. [DOI: 10.1517/14712598.2016.1158808] [Cited by in Crossref: 7] [Cited by in F6Publishing: 11] [Article Influence: 1.2] [Reference Citation Analysis]
42 Aravalli RN, Belcher JD, Steer CJ. Liver-targeted gene therapy: Approaches and challenges. Liver Transpl. 2015;21:718-737. [PMID: 25824605 DOI: 10.1002/lt.24122] [Cited by in Crossref: 19] [Cited by in F6Publishing: 20] [Article Influence: 3.2] [Reference Citation Analysis]
43 Marcos-Contreras OA, Smith SM, Bellinger DA, Raymer RA, Merricks E, Faella A, Pavani G, Zhou S, Nichols TC, High KA, Margaritis P. Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII. Blood 2016;127:565-71. [PMID: 26702064 DOI: 10.1182/blood-2015-09-671420] [Cited by in Crossref: 14] [Cited by in F6Publishing: 15] [Article Influence: 2.0] [Reference Citation Analysis]
44 Junge N, Mingozzi F, Ott M, Baumann U. Adeno-associated virus vector-based gene therapy for monogenetic metabolic diseases of the liver. J Pediatr Gastroenterol Nutr. 2015;60:433-440. [PMID: 25594875 DOI: 10.1097/mpg.0000000000000703] [Cited by in Crossref: 15] [Cited by in F6Publishing: 13] [Article Influence: 2.1] [Reference Citation Analysis]
45 Grimm D, Zolotukhin S. E Pluribus Unum: 50 Years of Research, Millions of Viruses, and One Goal--Tailored Acceleration of AAV Evolution. Mol Ther 2015;23:1819-31. [PMID: 26388463 DOI: 10.1038/mt.2015.173] [Cited by in Crossref: 66] [Cited by in F6Publishing: 65] [Article Influence: 9.4] [Reference Citation Analysis]
46 Rincon MY, VandenDriessche T, Chuah MK. Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation. Cardiovasc Res 2015;108:4-20. [PMID: 26239654 DOI: 10.1093/cvr/cvv205] [Cited by in Crossref: 86] [Cited by in F6Publishing: 85] [Article Influence: 12.3] [Reference Citation Analysis]
47 Arruda VR, Samelson-Jones BJ. Obstacles and future of gene therapy for hemophilia. Expert Opin Orphan Drugs 2015;3:997-1010. [PMID: 26900534 DOI: 10.1517/21678707.2015.1069179] [Cited by in Crossref: 19] [Cited by in F6Publishing: 21] [Article Influence: 2.7] [Reference Citation Analysis]
48 VandenDriessche T, Chuah MK. Moving forward toward a cure for hemophilia B. Mol Ther 2015;23:809-11. [PMID: 25943496 DOI: 10.1038/mt.2015.56] [Cited by in Crossref: 5] [Cited by in F6Publishing: 5] [Article Influence: 0.7] [Reference Citation Analysis]
49 Hastie E, Samulski RJ. Recombinant adeno-associated virus vectors in the treatment of rare diseases. Expert Opin Orphan Drugs 2015;3:675-89. [PMID: 27668135 DOI: 10.1517/21678707.2015.1039511] [Cited by in Crossref: 15] [Cited by in F6Publishing: 12] [Article Influence: 2.1] [Reference Citation Analysis]
50 Yao Y, Jin S, Long H, Yu Y, Zhang Z, Cheng G, Xu C, Ding Y, Guan Q, Li N, Fu S, Chen XJ, Yan YB, Zhang H, Tong P, Tan Y, Yu Y, Fu S, Li J, He GJ, Wu Q. RNAe: an effective method for targeted protein translation enhancement by artificial non-coding RNA with SINEB2 repeat. Nucleic Acids Res 2015;43:e58. [PMID: 25722369 DOI: 10.1093/nar/gkv125] [Cited by in Crossref: 20] [Cited by in F6Publishing: 19] [Article Influence: 2.9] [Reference Citation Analysis]
51 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: 14] [Article Influence: 1.7] [Reference Citation Analysis]
52 Aronovich EL, Hackett PB. Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier. Mol Genet Metab 2015;114:83-93. [PMID: 25410058 DOI: 10.1016/j.ymgme.2014.09.011] [Cited by in Crossref: 34] [Cited by in F6Publishing: 27] [Article Influence: 4.3] [Reference Citation Analysis]
53 Rincon MY, Sarcar S, Danso-Abeam D, Keyaerts M, Matrai J, Samara-Kuko E, Acosta-Sanchez A, Athanasopoulos T, Dickson G, Lahoutte T, De Bleser P, VandenDriessche T, Chuah MK. Genome-wide computational analysis reveals cardiomyocyte-specific transcriptional Cis-regulatory motifs that enable efficient cardiac gene therapy. Mol Ther 2015;23:43-52. [PMID: 25195597 DOI: 10.1038/mt.2014.178] [Cited by in Crossref: 25] [Cited by in F6Publishing: 26] [Article Influence: 3.1] [Reference Citation Analysis]
54 Hackett PB, Aronovich EL. Rational design for enhanced gene therapy with DNA transposons. Mol Ther 2014;22:1575-7. [PMID: 25186559 DOI: 10.1038/mt.2014.149] [Reference Citation Analysis]
55 Di Matteo M, Samara-Kuko E, Ward NJ, Waddington SN, McVey JH, Chuah MK, VandenDriessche T. Hyperactive piggyBac transposons for sustained and robust liver-targeted gene therapy. Mol Ther 2014;22:1614-24. [PMID: 25034357 DOI: 10.1038/mt.2014.131] [Cited by in Crossref: 38] [Cited by in F6Publishing: 39] [Article Influence: 4.8] [Reference Citation Analysis]