Publications

1. Cui M, Su Q, Yip M, McGowan J, Punzo C, Gao G, Tai PWL. The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2. Gene Ther. 2024 Aug 12. PMID: 39134629.
2. Liu H, Zhang Y, Yip M, Ren L, Liang J, Chen X, Liu N, Du A, Wang J, Chang H, Oh H, Zhou C, Xing R, Xu M, Guo P, Gessler D, Xie J, Tai PWL, Gao G, Wang D. Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection. Mol Ther Methods Clin Dev. 2024 Jun 13; 32(2):101230. PMID: 38558570.
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3. Xie Q, Chen X, Ma H, Zhu Y, Ma Y, Jalinous L, Cox GF, Weaver F, Yang J, Kennedy Z, Gruntman A, Du A, Su Q, He R, Tai PW, Gao G, Xie J. Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor. EMBO Mol Med. 2024 Apr; 16(4):945-965. PMID: 38413838.
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4. Tai PWL. Integration of Gene Therapy Vectors: A Risk Factor for Tumorigenesis or Another Commensal Property of Adeno-Associated Viruses That Benefits Long-Term Transgene Expression? Hum Gene Ther. 2023 Nov; 34(21-22):1074-1076. PMID: 37930948.
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5. Yip M, Chen J, Zhi Y, Tran NT, Namkung S, Pastor E, Gao G, Tai PWL. Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time Sequencing. Viruses. 2023 05 24; 15(6). PMID: 37376529.
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6. Tai PWL, Xie J. Learning to lock down genetic diseases without throwing away the key. Mol Ther. 2023 Jul 05; 31(7):1862-1863. PMID: 36933560.
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7. Namkung S, Tran NT, Manokaran S, He R, Su Q, Xie J, Gao G, Tai PWL. Direct ITR-to-ITR Nanopore Sequencing of AAV Vector Genomes. Hum Gene Ther. 2022 11; 33(21-22):1187-1196. PMID: 36178359.
   Citations: 4     
8. Tran NT, Lecomte E, Saleun S, Namkung S, Robin C, Weber K, Devine E, Blouin V, Adjali O, Ayuso E, Gao G, Penaud-Budloo M, Tai PWL. Human and Insect Cell-Produced Recombinant Adeno-Associated Viruses Show Differences in Genome Heterogeneity. Hum Gene Ther. 2022 04; 33(7-8):371-388. PMID: 35293222.
   Citations: 20    
9. Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, Douthwright C, Gernoux G, Blackwood M, Mueller C, Tai PWL, Jiang X, Bateman S, Spanakis SG, Parzych J, Keeler AM, Abayazeed A, Rohatgi S, Gibson L, Finberg R, Barton BA, Vardar Z, Shazeeb MS, Gounis M, Tifft CJ, Eichler FS, Brown RH, Martin DR, Gray-Edwards HL, Sena-Esteves M. AAV gene therapy for Tay-Sachs disease. Nat Med. 2022 02; 28(2):251-259. PMID: 35145305.
   Citations: 28     
10. Ibraheim R, Tai PWL, Mir A, Javeed N, Wang J, Rodr?guez TC, Namkung S, Nelson S, Khokhar ES, Mintzer E, Maitland S, Chen Z, Cao Y, Tsagkaraki E, Wolfe SA, Wang D, Pai AA, Xue W, Gao G, Sontheimer EJ. Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo. Nat Commun. 2021 11 01; 12(1):6267. PMID: 34725353.
    Citations: 31     
11. Pan X, Yue Y, Boftsi M, Wasala LP, Tran NT, Zhang K, Pintel DJ, Tai PWL, Duan D. Rational engineering of a functional CpG-free ITR for AAV gene therapy. Gene Ther. 2022 06; 29(6):333-345. PMID: 34611321.
    Citations: 15     
12. Cheng SY, Luo Y, Malachi A, Ko J, Su Q, Xie J, Tian B, Lin H, Ke X, Zheng Q, Tai PWL, Gao G, Punzo C. Low-Dose Recombinant Adeno-Associated Virus-Mediated Inhibition of Vascular Endothelial Growth Factor Can Treat Neovascular Pathologies Without Inducing Retinal Vasculitis. Hum Gene Ther. 2021 07; 32(13-14):649-666. PMID: 34182803.
    Citations: 3     
13. Su W, Sun S, Tian B, Tai PWL, Luo Y, Ko J, Zhan W, Ke X, Zheng Q, Li X, Yan H, Gao G, Lin H. Efficacious, safe, and stable inhibition of corneal neovascularization by AAV-vectored anti-VEGF therapeutics. Mol Ther Methods Clin Dev. 2021 Sep 10; 22:107-121. PMID: 34514023.
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14. Zhan W, Muhuri M, Tai PWL, Gao G. Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens? Front Immunol. 2021; 12:673699. PMID: 34046041.
    Citations: 12     
15. Muhuri M, Zhan W, Maeda Y, Li J, Lotun A, Chen J, Sylvia K, Dasgupta I, Arjomandnejad M, Nixon T, Keeler AM, Manokaran S, He R, Su Q, Tai PWL, Gao G. Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction. Front Immunol. 2021; 12:674242. PMID: 33995418.
    Citations: 9     
16. Qin W, Xu G, Tai PWL, Wang C, Luo L, Li C, Hu X, Xue J, Lu Y, Zhou Q, Wei Q, Wen T, Hu J, Xiao Y, Yang L, Li W, Flotte TR, Wei Y, Gao G. Large-scale molecular epidemiological analysis of AAV in a cancer patient population. Oncogene. 2021 Apr; 40(17):3060-3071. PMID: 33782545.
    Citations: 5     
17. Chan YK, Wang SK, Chu CJ, Copland DA, Letizia AJ, Costa Verdera H, Chiang JJ, Sethi M, Wang MK, Neidermyer WJ, Chan Y, Lim ET, Graveline AR, Sanchez M, Boyd RF, Vihtelic TS, Inciong RGCO, Slain JM, Alphonse PJ, Xue Y, Robinson-McCarthy LR, Tam JM, Jabbar MH, Sahu B, Adeniran JF, Muhuri M, Tai PWL, Xie J, Krause TB, Vernet A, Pezone M, Xiao R, Liu T, Wang W, Kaplan HJ, Gao G, Dick AD, Mingozzi F, McCall MA, Cepko CL, Church GM. Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses. Sci Transl Med. 2021 02 10; 13(580). PMID: 33568518.
    Citations: 67     
18. Bulcha JT, Wang Y, Ma H, Tai PWL, Gao G. Viral vector platforms within the gene therapy landscape. Signal Transduct Target Ther. 2021 02 08; 6(1):53. PMID: 33558455.
    Citations: 263     
19. Muhuri M, Maeda Y, Ma H, Ram S, Fitzgerald KA, Tai PW, Gao G. Overcoming innate immune barriers that impede AAV gene therapy vectors. J Clin Invest. 2021 01 04; 131(1). PMID: 33393506.
    Citations: 44     
20. Tran NT, Heiner C, Weber K, Weiand M, Wilmot D, Xie J, Wang D, Brown A, Manokaran S, Su Q, Zapp ML, Gao G, Tai PWL. AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity. Mol Ther Methods Clin Dev. 2020 Sep 11; 18:639-651. PMID: 32775498.
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21. Hsu HL, Brown A, Loveland AB, Lotun A, Xu M, Luo L, Xu G, Li J, Ren L, Su Q, Gessler DJ, Wei Y, Tai PWL, Korostelev AA, Gao G. Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties. Nat Commun. 2020 06 30; 11(1):3279. PMID: 32606306.
    Citations: 20     
22. Yang YS, Xie J, Chaugule S, Wang D, Kim JM, Kim J, Tai PWL, Seo SK, Gravallese E, Gao G, Shim JH. Bone-Targeting AAV-Mediated Gene Silencing in Osteoclasts for Osteoporosis Therapy. Mol Ther Methods Clin Dev. 2020 Jun 12; 17:922-935. PMID: 32405514.
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23. Tai PWL. ITRs: The Terminal Frontier. Hum Gene Ther. 2020 02; 31(3-4):143-144. PMID: 32073925.
    Citations: 1      
24. Xie J, Tai PWL, Brown A, Gong S, Zhu S, Wang Y, Li C, Colpan C, Su Q, He R, Ma H, Li J, Ye H, Ko J, Zamore PD, Gao G. Effective and Accurate Gene Silencing by a Recombinant AAV-Compatible MicroRNA Scaffold. Mol Ther. 2020 02 05; 28(2):422-430. PMID: 31843447.
    Citations: 11    
25. Taghian T, Marosfoi MG, Puri AS, Cataltepe OI, King RM, Diffie EB, Maguire AS, Martin DR, Fernau D, Batista AR, Kuchel T, Christou C, Perumal R, Chandra S, Gamlin PD, Bertrand SG, Flotte TR, McKenna-Yasek D, Tai PWL, Aronin N, Gounis MJ, Sena-Esteves M, Gray-Edwards HL. A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna. Mol Ther. 2020 02 05; 28(2):411-421. PMID: 31813800.
    Citations: 39    
26. Wang D, Niu Y, Ren L, Kang Y, Tai PWL, Si C, Mendonca CA, Ma H, Gao G, Ji W. Gene Delivery to Nonhuman Primate Preimplantation Embryos Using Recombinant Adeno-Associated Virus. Adv Sci (Weinh). 2019 Nov 06; 6(21):1900440. PMID: 31728271.
    Citations: 7        
27. Yang YS, Xie J, Wang D, Kim JM, Tai PWL, Gravallese E, Gao G, Shim JH. Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis. Nat Commun. 2019 07 04; 10(1):2958. PMID: 31273195.
    Citations: 48     
28. Xu M, Li J, Xie J, He R, Su Q, Gao G, Tai PWL. High-Throughput Quantification of In Vivo Adeno-Associated Virus Transduction with Barcoded Non-Coding RNAs. Hum Gene Ther. 2019 08; 30(8):946-956. PMID: 31072208.
    Citations: 3     
29. Xiao Y, Muhuri M, Li S, Qin W, Xu G, Luo L, Li J, Letizia AJ, Wang SK, Chan YK, Wang C, Fuchs SP, Wang D, Su Q, Nahid MA, Church GM, Farzan M, Yang L, Wei Y, Desrosiers RC, Mueller C, Tai PW, Gao G. Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity. JCI Insight. 2019 05 21; 5. PMID: 31112525.
    Citations: 19    
30. Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019 05; 18(5):358-378. PMID: 30710128.
    Citations: 698     
31. Gessler DJ, Tai PWL, Li J, Gao G. Intravenous Infusion of AAV for Widespread Gene Delivery to the Nervous System. Methods Mol Biol. 2019; 1950:143-163. PMID: 30783972.
    Citations: 14     
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 10; 36(9):839-842. PMID: 30102296.
    Citations: 24    
33. Tai PWL, Xie J, Fong K, Seetin M, Heiner C, Su Q, Weiand M, Wilmot D, Zapp ML, Gao G. Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras. Mol Ther Methods Clin Dev. 2018 Jun 15; 9:130-141. PMID: 29766023.
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34. Yoon Y, Wang D, Tai PWL, Riley J, Gao G, Rivera-P?rez JA. Streamlined ex vivo and in vivo genome editing in mouse embryos using recombinant adeno-associated viruses. Nat Commun. 2018 01 29; 9(1):412. PMID: 29379011.
    Citations: 40     
35. Lu Y, Tai PWL, Ai J, Gessler DJ, Su Q, Yao X, Zheng Q, Zamore PD, Xu X, Gao G. Transcriptome Profiling of Neovascularized Corneas Reveals miR-204 as a Multi-target Biotherapy Deliverable by rAAVs. Mol Ther Nucleic Acids. 2018 Mar 02; 10:349-360. PMID: 29499946.
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36. Tai PWL, Wu H, van Wijnen AJ, Stein GS, Stein JL, Lian JB. Genome-wide DNase hypersensitivity, and occupancy of RUNX2 and CTCF reveal a highly dynamic gene regulome during MC3T3 pre-osteoblast differentiation. PLoS One. 2017; 12(11):e0188056. PMID: 29176792.
    Citations: 5     
37. Xie J, Mao Q, Tai PWL, He R, Ai J, Su Q, Zhu Y, Ma H, Li J, Gong S, Wang D, Gao Z, Li M, Zhong L, Zhou H, Gao G. Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity. Mol Ther. 2017 06 07; 25(6):1363-1374. PMID: 28462820.
    Citations: 44     
38. Ai J, Ibraheim R, Tai PWL, Gao G. A Scalable and Accurate Method for Quantifying Vector Genomes of Recombinant Adeno-Associated Viruses in Crude Lysate. Hum Gene Ther Methods. 2017 06; 28(3):139-147. PMID: 28488944.
    Citations: 13     
39. Wu H, Gordon JA, Whitfield TW, Tai PW, van Wijnen AJ, Stein JL, Stein GS, Lian JB. Chromatin dynamics regulate mesenchymal stem cell lineage specification and differentiation to osteogenesis. Biochim Biophys Acta Gene Regul Mech. 2017 Apr; 1860(4):438-449. PMID: 28077316.
    Citations: 34     
40. Carr FE, Tai PW, Barnum MS, Gillis NE, Evans KG, Taber TH, White JH, Tomczak JA, Jaworski DM, Zaidi SK, Lian JB, Stein JL, Stein GS. Thyroid Hormone Receptor-? (TR?) Mediates Runt-Related Transcription Factor 2 (Runx2) Expression in Thyroid Cancer Cells: A Novel Signaling Pathway in Thyroid Cancer. Endocrinology. 2016 08; 157(8):3278-92. PMID: 27253998.
    Citations: 18     
41. Li K, Zhang H, Qiu J, Lin Y, Liang J, Xiao X, Fu L, Wang F, Cai J, Tan Y, Zhu W, Yin W, Lu B, Xing F, Tang L, Yan M, Mai J, Li Y, Chen W, Qiu P, Su X, Gao G, Tai PW, Hu J, Yan G. Activation of Cyclic Adenosine Monophosphate Pathway Increases the Sensitivity of Cancer Cells to the Oncolytic Virus M1. Mol Ther. 2016 Feb; 24(1):156-65. PMID: 26373347.
    Citations: 25     
42. Barutcu AR, Tai PW, Wu H, Gordon JA, Whitfield TW, Dobson JR, Imbalzano AN, Lian JB, van Wijnen AJ, Stein JL, Stein GS. The bone-specific Runx2-P1 promoter displays conserved three-dimensional chromatin structure with the syntenic Supt3h promoter. Nucleic Acids Res. 2014; 42(16):10360-72. PMID: 25120271.
    Citations: 22     
43. Tai PW, Wu H, Gordon JA, Whitfield TW, Barutcu AR, van Wijnen AJ, Lian JB, Stein GS, Stein JL. Epigenetic landscape during osteoblastogenesis defines a differentiation-dependent Runx2 promoter region. Gene. 2014 Oct 15; 550(1):1-9. PMID: 24881813.
    Citations: 18     
44. Tai PW, Zaidi SK, Wu H, Grandy RA, Montecino M, van Wijnen AJ, Lian JB, Stein GS, Stein JL. The dynamic architectural and epigenetic nuclear landscape: developing the genomic almanac of biology and disease. J Cell Physiol. 2014 Jun; 229(6):711-27. PMID: 24242872.
    Citations: 9     
45. Wu H, Whitfield TW, Gordon JA, Dobson JR, Tai PW, van Wijnen AJ, Stein JL, Stein GS, Lian JB. Genomic occupancy of Runx2 with global expression profiling identifies a novel dimension to control of osteoblastogenesis. Genome Biol. 2014 Mar 21; 15(3):R52. PMID: 24655370.
    Citations: 83     
46. Tsubota Y, Frey JM, Tai PW, Welikson RE, Raines EW. Monocyte ADAM17 promotes diapedesis during transendothelial migration: identification of steps and substrates targeted by metalloproteinases. J Immunol. 2013 Apr 15; 190(8):4236-44. PMID: 23479224.
    Citations: 18   
47. Himeda CL, Tai PW, Hauschka SD. Analysis of muscle gene transcription in cultured skeletal muscle cells. Methods Mol Biol. 2012; 798:425-43. PMID: 22130852.
48. Tai PW, Smith CL, Angello JC, Hauschka SD. Analysis of fiber-type differences in reporter gene expression of b-gal transgenic muscle. Methods Mol Biol. 2012; 798:445-59. PMID: 22130853.
    Citations: 2   
49. Tai PW, Fisher-Aylor KI, Himeda CL, Smith CL, Mackenzie AP, Helterline DL, Angello JC, Welikson RE, Wold BJ, Hauschka SD. Differentiation and fiber type-specific activity of a muscle creatine kinase intronic enhancer. Skelet Muscle. 2011 Jul 07; 1:25. PMID: 21797989.
    Citations: 18        
50. Salva MZ, Himeda CL, Tai PW, Nishiuchi E, Gregorevic P, Allen JM, Finn EE, Nguyen QG, Blankinship MJ, Meuse L, Chamberlain JS, Hauschka SD. Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle. Mol Ther. 2007 Feb; 15(2):320-9. PMID: 17235310.
    Citations: 121