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Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances Expression of Transgenes Delivered by Retroviral Vectors

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Enhanced Expression of Transgenes from Adeno-Associated Virus Vectors with the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element: Implications for Gene Therapy

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Malignant transformation of immortalized transgenic hepatocytes after transfection with hepatitis B virus DNA.

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Human hepatitis virus X gene encodes a regulatory domain that represses transactivation of X protein.

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Induction of apoptosis by the transactivating domains of the hepatitis B virus X gene leads to suppression of oncogenic transformation of primary rat embryo fibroblasts

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Long-term replacement of a mutated nonfunctional CNS gene: reversal of hypothalamic diabetes insipidus using an EIAV-based lentiviral vector expressing arginine vasopressin

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Neuroprotection in a rat Parkinson model by GDNF gene therapy using EIAV vector

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Analysis of gene transfer and expression in skeletal muscle using enhanced EIAV lentivirus vectors

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Woodchuck Hepatitis Virus Enhancer I and Enhancer II Are Both Involved in N- myc2 Activation in Woodchuck Liver Tumors

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Hepatocyte‐specific gene expression from integrated lentiviral vectors

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Adeno-associated virus vector as a platform for gene therapy delivery

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Adeno-associated virus as a delivery vector for gene therapy of human diseases

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Gene delivery by lentivirus vectors

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Lentiviral vectors: basic to translational

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Gene Therapy Progress and Prospects: Development of improved lentiviral and retroviral vectors – design, biosafety, and production

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Oncogenesis Following Delivery of a Nonprimate Lentiviral Gene Therapy Vector to Fetal and Neonatal Mice

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Successful Reconstitution of Immunity in ADA-SCID by Stem Cell Gene Therapy Following Cessation of PEG-ADA and Use of Mild Preconditioning

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Human Mesenchymal Stem Cells Genetically Engineered to Overexpress Brain-derived Neurotrophic Factor Improve Outcomes in Huntington's Disease Mouse Models

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Lentiviral delivery of short hairpin RNAs

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Upscaling of lentiviral vector production by tangential flow filtration

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Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids

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Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression

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No Evidence for Tumorigenesis of AAV Vectors in a Large-Scale Study in Mice

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Mild Hypothermia Improves Transient Gene Expression Yields Several Fold in Chinese Hamster Ovary Cells

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Retrovirus Vectors: Toward the Plentivirus?

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Lentiviral Vectors: Their Molecular Design, Safety, and Use in Laboratory and Preclinical Research

Dropulić, B. (2011) · Human Gene Therapy · doi:10.1089/hum.2011.058 · Cited by 106 · see citing works

Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo

Tolmachova, T., Tolmachov, O. E., Barnard, A. R., Silva, S. R. D., Lipinski, D. M., Walker, N. J., MacLaren, R. E., & Seabra, M. C. (2013) · Journal of Molecular Medicine · doi:10.1007/s00109-013-1006-4 · Cited by 92 · see citing works

Therapeutic potential of CERE-110 (AAV2-NGF): Targeted, stable, and sustained NGF delivery and trophic activity on rodent basal forebrain cholinergic neurons

Bishop, K. M., Hofer, E. K., Mehta, A., Ramirez, A., Sun, L., Tuszynski, M. H., & Bartus, R. T. (2008) · Experimental Neurology · doi:10.1016/j.expneurol.2008.03.004 · Cited by 91 · see citing works

The Genetic Engineering of Hematopoietic Stem Cells: the Rise of Lentiviral Vectors, the Conundrum of the LTR, and the Promise of Lineage-restricted Vectors

Chang, A. H., & Sadelain, M. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300060 · Cited by 88 · see citing works

Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS

Zanta, M.-A., Charrier, S., Brice-Ouzet, A., Martin, S., Opolon, P., Thrasher, A. J., Hope, T. J., & Galy, A. H. M. (2009) · Gene Therapy · doi:10.1038/gt.2009.3 · Cited by 86 · see citing works

Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer

Ding, Z., Georgiev, P., & Thöny, B. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302684 · Cited by 85 · see citing works

Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott–Aldrich Syndrome in Preclinical Models

Marangoni, F., Bosticardo, M., Charrier, S., Draghici, E., Locci, M., Scaramuzza, S., Panaroni, C., Ponzoni, M., Sanvito, F., Doglioni, C., et al. (2009) · Molecular Therapy · doi:10.1038/mt.2009.31 · Cited by 85 · see citing works

Gene therapy progress and prospects: Duchenne muscular dystrophy

Foster, K., Foster, H., & Dickson, J. G. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302877 · Cited by 72 · see citing works

Development of a Wilms' tumor antigen-specific T-cell receptor for clinical trials: engineered patient's T cells can eliminate autologous leukemia blasts in NOD/SCID mice

Xue, S., Gao, L., Thomas, S., Hart, D. P., Xue, J. Z., Gillmore, R., Voss, R.-H., Morris, E. C., & Stauss, H. J. (2009) · Haematologica · doi:10.3324/haematol.2009.006486 · Cited by 61 · see citing works

EIAV vector-mediated delivery of endostatin or angiostatin inhibits angiogenesis and vascular hyperpermeability in experimental CNV

Balaggan, K. S., Binley, K. E., Esapa, M., MacLaren, R. E., Iqball, S., Durán, Y., Pearson, R. A., Kan, O., Barker, S. E., Smith, A. J., et al. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302769 · Cited by 57 · see citing works

Generation of multi-functional antigen-specific human T-cells by lentiviral TCR gene transfer

Perro, M., Tsang, J. Y., Xue, S., Escors, D., Cesco-Gaspere, M., Pospori, C., Gao, L., Hart, D. P., Collins, M. K., Stauss, H. J., et al. (2010) · Gene Therapy · doi:10.1038/gt.2010.4 · Cited by 51 · see citing works

Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review

Wang, J.-H., Zhan, W., Gallagher, T. L., & Gao, G. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.10.017 · Cited by 48 · see citing works

Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside

Xu, L., Yao, S., Ding, Y. E., Xie, M., Feng, D., Sha, P., Tan, L., Bei, F., & Yao, Y. (2024) · Journal of Translational Medicine · doi:10.1186/s12967-024-05661-2 · Cited by 48 · see citing works

Development of Lentiviral Vectors with Optimized Transcriptional Activity for the Gene Therapy of Patients with Fanconi Anemia

Gónzalez-Murillo, Á., Lozano, M. L., Alvarez, L., Jacome, A., Almarza, E., Navarro, S., Segovia, J., Hanenberg, H., Guenechea, G., Bueren, J., et al. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.141 · Cited by 54 · see citing works

Designing Plasmid Vectors

Tolmachov, O. E. (2009) · Methods in molecular biology · doi:10.1007/978-1-59745-561-9_6 · Cited by 43 · see citing works

Sustained correction of OTC deficiency in spfash mice using optimized self-complementary AAV2/8 vectors

Wang, L., Wang, H., Morizono, H., Bell, P. L., Jones, D., Lin, J., McMenamin, D., Yu, H., Batshaw, M. L., & Wilson, J. M. (2011) · Gene Therapy · doi:10.1038/gt.2011.111 · Cited by 41 · see citing works

Delivering genes with human immunodeficiency virus-derived vehicles: still state-of-the-art after 25 years

Wolff, J. H., & Mikkelsen, J. G. (2022) · Journal of Biomedical Science · doi:10.1186/s12929-022-00865-4 · Cited by 41 · see citing works

Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector

Becker, P. S., Taylor, J. A., Trobridge, G. D., Zhao, X., Beard, B. C., Chien, S. S., Adair, J. E., Kohn, D. B., Wagner, J. E., Shimamura, A., et al. (2010) · Gene Therapy · doi:10.1038/gt.2010.62 · Cited by 38 · see citing works

The CD20/αCD20 ‘suicide’ system: novel vectors with improved safety and expression profiles and efficient elimination of CD20-transgenic T cells

van Meerten, T., Claessen, M.-J., Hagenbeek, A., & Ebeling, S. B. (2006) · Gene Therapy · doi:10.1038/sj.gt.3302705 · Cited by 38 · see citing works

Lentiviral Vectors for T-cell Suicide Gene Therapy: Preservation of T-cell Effector Function After Cytokine-mediated Transduction

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Lentivirus-mediated gene transfer to the rat, ovine and human cornea

Parker, D. G., Kaufmann, C., Brereton, H. M., Anson, D. S., Francis-Staite, L., Jessup, C. F., Marshall, K., Tan, C., Koldej, R. M., Coster, D. J., et al. (2007) · Gene Therapy · doi:10.1038/sj.gt.3302921 · Cited by 35 · see citing works

Viral vectors for gene delivery to the inner ear

Maguire, C. A., & Corey, D. P. (2020) · Hearing Research · doi:10.1016/j.heares.2020.107927 · Cited by 32 · see citing works

Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A

Johnston, J. M., Denning, G., Doering, C. B., & Spencer, H. T. (2012) · Gene Therapy · doi:10.1038/gt.2012.76 · Cited by 34 · see citing works

Hematopoietic Stem Cell Gene Therapy for Cystinosis: From Bench-to-Bedside

Cherqui, S. (2021) · Cells · doi:10.3390/cells10123273 · Cited by 33 · see citing works

Strategies to improve safety profile of AAV vectors

Suoranta, T., Laham-Karam, N., & Ylä‐Herttuala, S. (2022) · Frontiers in Molecular Medicine · doi:10.3389/fmmed.2022.1054069 · Cited by 30 · see citing works

Neonatal Gene Therapy of Glycogen Storage Disease Type Ia Using a Feline Immunodeficiency Virus–based Vector

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Sustained Expression from DNA Vectors

Wong, S. P., Argyros, O., & Harbottle, R. P. (2014) · Advances in genetics · doi:10.1016/bs.adgen.2014.11.002 · Cited by 26 · see citing works

New Strategies for Cardiovascular Gene Therapy: Regulatable Pre-Emptive Expression of Pro-Angiogenic and Antioxidant Genes

Dulak, J., Zagórska, A., Wegiel, B., Łoboda, A., & Józkowicz, A. (2006) · Cell Biochemistry and Biophysics · doi:10.1385/cbb:44:1:031 · Cited by 24 · see citing works

Specificity for the tumor-associated self-antigen WT1 drives the development of fully functional memory T cells in the absence of vaccination

Pospori, C., Xue, S., Holler, A., Voisine, C., Perro, M., King, J. P., Fallah‐Arani, F., Flutter, B. R., Chakraverty, R. K., Stauss, H. J., et al. (2011) · Blood · doi:10.1182/blood-2010-08-304568 · Cited by 23 · see citing works

Metabolic Correction of Congenital Erythropoietic Porphyria with iPSCs Free of Reprogramming Factors

Bedel, A., Taillepierre, M., Guyonnet‐Dupérat, V., Lippert, É., Dubus, P. R., Dabernat, S., Mautuit, T., Cardinaud, B., Pain, C., Rousseau, B., et al. (2012) · The American Journal of Human Genetics · doi:10.1016/j.ajhg.2012.05.026 · Cited by 22 · see citing works

The Fetal Mouse Is a Sensitive Genotoxicity Model That Exposes Lentiviral-associated Mutagenesis Resulting in Liver Oncogenesis

Nowrouzi, A., Cheung, W. T., Li, T., Zhang, X., Arens, A., Paruzynski, A., Waddington, S. N., Osejindu, E., Reja, S., Kalle, C. V., et al. (2013) · Molecular Therapy · doi:10.1038/mt.2012.224 · Cited by 22 · see citing works

Recombinant AAV Vectors for Enhanced Expression of Authentic IgG

Fuchs, S. P., Martinez-Navío, J. M., Gao, G., & Desrosiers, R. C. (2016) · PLoS ONE · doi:10.1371/journal.pone.0158009 · Cited by 21 · see citing works

Ethics of iPSC-Based Clinical Research for Age-Related Macular Degeneration: Patient-Centered Risk-Benefit Analysis

Nakano‐Okuno, M., Borah, B. R., & Nakano, I. (2014) · Stem Cell Reviews and Reports · doi:10.1007/s12015-014-9536-x · Cited by 22 · see citing works

Impact of Age at Administration, Lysosomal Storage, and Transgene Regulatory Elements on AAV2/8-Mediated Rat Liver Transduction

Cotugno, G., Annunziata, P., Barone, M. V., Karali, M., Banfi, S., & Auricchio, A. (2012) · PLoS ONE · doi:10.1371/journal.pone.0033286 · Cited by 20 · see citing works

Differential Transgene Expression Profiles in Rat Brain, Using rAAV2/1 Vectors with Tetracycline-Inducible and Cytomegalovirus Promoters

Bockstael, O., Chtarto, A., Wakkinen, J., Yang, X. L., Melas, C., Levivier, M., Brotchi, J., & Tenenbaum, L. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.099 · Cited by 20 · see citing works

Vector design for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells

Schambach, A., & Baum, C. (2007) · DNA repair · doi:10.1016/j.dnarep.2007.03.017 · Cited by 18 · see citing works

Construction of a novel expression cassette for increasing transgene expression in vivo in endothelial cells of large blood vessels

Dronadula, N., Du, L., Flynn, R., Buckler, J., Kho, J., Jiang, Z.-G., Tanaka, S., & Dichek, D. A. (2010) · Gene Therapy · doi:10.1038/gt.2010.173 · Cited by 18 · see citing works

New Vectors and Strategies for Cardiovascular Gene Therapy

Jaźwa, A., Józkowicz, A., & Dulak, J. (2007) · Current Gene Therapy · doi:10.2174/156652307779940243 · Cited by 18 · see citing works

Adeno-Associated Virus-Mediated Expression of Vascular Endothelial Growth Factor Peptides Inhibits Retinal Neovascularization in a Mouse Model of Oxygen-Induced Retinopathy

Deng, W., Yan, Z., Dinculescu, A., Pang, J., Teusner, J. T., Cortez, N. G., Berns, K. I., & Hauswirth, W. W. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.1247 · Cited by 15 · see citing works

Lentiviral Vectors: From Wild-Type Viruses to Efficient Multi-Functional Delivery Vectors

Arrasate, A., Lopez-Robles, C., Zuazo, M., Baños-Mateos, S., Martı́n, C., Lamsfus‐Calle, A., & Fertin, M. J. (2025) · International Journal of Molecular Sciences · doi:10.3390/ijms26178497 · Cited by 14 · see citing works

Elements of lentiviral vector design toward gene therapy for treating mucopolysaccharidosis I

Ou, L., Przybilla, M. J., Koniar, B. L., & Whitley, C. B. (2016) · Molecular Genetics and Metabolism Reports · doi:10.1016/j.ymgmr.2015.11.004 · Cited by 14 · see citing works

Improved microRNA suppression by WPRE-linked tough decoy microRNA sponges

Hollensen, A. K., Thomsen, R., Bak, R. O., Petersen, C. C., Ermegaard, E. R., Aagaard, L., Damgaard, C. K., & Mikkelsen, J. G. (2017) · RNA · doi:10.1261/rna.061192.117 · Cited by 13 · see citing works

Targeting adenoviral transgene expression to neurons

Sims, K., Ahmed, Z., Gonzalez, A. M., Read, M. L., Cooper‐Charles, L., Berry, M., & Logan, A. (2008) · Molecular and Cellular Neuroscience · doi:10.1016/j.mcn.2008.07.020 · Cited by 11 · see citing works

Adeno-Associated Viral Gene Therapy for Retinal Disorders

Silva, S. R. D., McClements, M. E., Hankins, M. W., & MacLaren, R. E. (2015) · Neuromethods · doi:10.1007/978-1-4939-2306-9_9 · Cited by 10 · see citing works

Modeling of congenital erythropoietic porphyria by RNA interference: a new tool for preclinical gene therapy evaluation

Richard, É., Lalanne, M., Lamrissi‐Garcia, I., Guyonnet‐Dupérat, V., Richard, E., Pitard, V., Mazurier, F., Moreau‐Gaudry, F., Ged, C., & de Verneuil, H. (2010) · The Journal of Gene Medicine · doi:10.1002/jgm.1478 · Cited by 10 · see citing works

Murine leukemia following irradiation conditioning for transplantation of lentivirally‐modified hematopoietic stem cells

Siapati, E. K., Bigger, B., Kashofer, K., Themis, M., Thrasher, A. J., & Bonnet, D. A. (2007) · European Journal Of Haematology · doi:10.1111/j.1600-0609.2006.00813.x · Cited by 9 · see citing works

Comparison of toxicogenomic profiles of two murine strains treated with HIV-1-based vectors for gene therapy

Zhao, Y., Keating, K., & Thorpe, R. (2007) · Toxicology and Applied Pharmacology · doi:10.1016/j.taap.2007.08.008 · Cited by 8 · see citing works

Optimization of adeno-associated viral (AAV) gene therapies vectors for balancing efficacy, longevity and safety for clinical application

Mehta, N., Gilbert, R., Chahal, P. S., Moreno, M., Nassoury, N., Coulombe, N., Gingras, R., Mullick, A., Drouin, S., Sasseville, M., et al. (2025) · Gene Therapy · doi:10.1038/s41434-025-00524-x · Cited by 7 · see citing works

Transcription Factor Binding Site in Promoter Determines the Pattern of Plasmid-Based Transgene Expression In Vivo

Zhang, C., & Liu, D. (2024) · Pharmaceutics · doi:10.3390/pharmaceutics16040544 · Cited by 6 · see citing works

Lentivirus vector driven by polybiquitin C promoter without woodchuck posttranscriptional regulatory element and central polypurine tract generates low level and short-lived reporter gene expression

Ngai, S. C., Rosli, R., Nordin, N., Veerakumarasivam, A., & Abdullah, S. (2012) · Gene · doi:10.1016/j.gene.2012.01.071 · Cited by 5 · see citing works

Ex Vivo Gene Therapy

Baranyi, L., Slepushkin, V., & Dropulić, B. (2013) · Elsevier eBooks · doi:10.1016/b978-0-12-394295-1.00001-9 · Cited by 4 · see citing works

Tetradecanoylphorbol-13-acetate (TPA) significantly increases AAV2/5 transduction of human neuronal cells in vitro

You, Q. S., Brown, L. A., McClements, M. E., Hankins, M. W., & MacLaren, R. E. (2011) · Experimental Eye Research · doi:10.1016/j.exer.2011.12.009 · Cited by 4 · see citing works

Development of VNX-101, an adeno-associated virus with less immunogenicity and efficient long-term expression of a CD19 T cell engager

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Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations

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