| scholarly article | Q13442814 |
| review article | Q7318358 |
| P2093 | author name string | Steven James Gray | |
| Ricardo Rivera-Soto | |||
| Sara Kathleen Powell | |||
| P2860 | cites work | Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2 | Q22337290 |
| The simian virus 40 small-t intron, present in many common expression vectors, leads to aberrant splicing | Q24634564 | ||
| Developments in the treatment of hemophilia B: focus on emerging gene therapy | Q26823376 | ||
| Quantitative comparison of constitutive promoters in human ES cells | Q28475300 | ||
| siRNAs can function as miRNAs | Q29615070 | ||
| Adeno-associated virus serotype 9 efficiently targets ischemic skeletal muscle following systemic delivery. | Q30414524 | ||
| Regulation of GluR2 promoter activity by neurotrophic factors via a neuron-restrictive silencer element | Q30866082 | ||
| Adeno-associated virus serotypes: vector toolkit for human gene therapy | Q33249130 | ||
| Comparison of insulators and promoters for expression of the Wiskott-Aldrich syndrome protein using lentiviral vectors | Q33408529 | ||
| MECP2 isoform-specific vectors with regulated expression for Rett syndrome gene therapy | Q33497321 | ||
| Transgenes delivered by lentiviral vector are suppressed in human embryonic stem cells in a promoter-dependent manner | Q33570141 | ||
| Systematic comparison of constitutive promoters and the doxycycline-inducible promoter | Q33582194 | ||
| Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. | Q33643620 | ||
| A shortened adeno-associated virus expression cassette for CFTR gene transfer to cystic fibrosis airway epithelia. | Q33864423 | ||
| Long-term reproducible expression in human fetal liver hematopoietic stem cells with a UCOE-based lentiviral vector | Q34035474 | ||
| Adeno-associated virus vectorology, manufacturing, and clinical applications | Q34172314 | ||
| Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer | Q34516810 | ||
| Insights into glutamate transport regulation in human astrocytes: cloning of the promoter for excitatory amino acid transporter 2 (EAAT2). | Q34762681 | ||
| Self-complementary AAV vectors; advances and applications | Q34804660 | ||
| Engineered lentiviral vector targeting astrocytes in vivo | Q34862612 | ||
| Intravitreal injection of AAV2 transduces macaque inner retina | Q34937075 | ||
| Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors | Q35226125 | ||
| Gene therapy progress and prospects: transcription regulatory systems. | Q35676294 | ||
| Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease | Q35988683 | ||
| A generic intron increases gene expression in transgenic mice | Q36695893 | ||
| Definition of the upstream efficiency element of the simian virus 40 late polyadenylation signal by using in vitro analyses | Q36706227 | ||
| Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells | Q37069729 | ||
| Recent advances in lentiviral vector development and applications | Q37678176 | ||
| Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons | Q37684186 | ||
| State-of-the-art gene-based therapies: the road ahead | Q37862074 | ||
| MicroRNA therapeutics. | Q37869804 | ||
| Recent gene therapy advancements for neurological diseases. | Q38085469 | ||
| Optimizing retroviral gene expression for effective therapies | Q38091750 | ||
| New developments in lentiviral vector design, production and purification | Q38099382 | ||
| Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element. | Q39580820 | ||
| Desmin-regulated lentiviral vectors for skeletal muscle gene transfer | Q39644545 | ||
| Optimal promoter usage for lentiviral vector-mediated transduction of cultured central nervous system cells. | Q39721811 | ||
| Gene delivery to the juvenile mouse liver using AAV2/8 vectors | Q39991575 | ||
| Role of the hepatitis B virus posttranscriptional regulatory element in export of intronless transcripts | Q40016714 | ||
| Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose | Q40039645 | ||
| Hepatitis B virus RNA element that facilitates accumulation of surface gene transcripts in the cytoplasm. | Q40040638 | ||
| Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors | Q40150079 | ||
| CMV enhancer/human PDGF-beta promoter for neuron-specific transgene expression | Q40606300 | ||
| Gene transduction efficiency in cells of different species by HIV and EIAV vectors | Q40722101 | ||
| Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1alpha promoter. | Q40769266 | ||
| Systematic determination of the packaging limit of lentiviral vectors | Q40776442 | ||
| Lentiviral vectors for enhanced gene expression in human hematopoietic cells | Q40841789 | ||
| Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: implications for gene therapy | Q40924169 | ||
| Gene transfer and expression in oligodendrocytes under the control of myelin basic protein transcriptional control region mediated by adeno-associated virus | Q41052074 | ||
| Role of intron I in expression of the human factor IX gene | Q41360681 | ||
| Long-term AAV vector gene and protein expression in mouse brain from a small pan-cellular promoter is similar to neural cell promoters | Q42199204 | ||
| A compact dual promoter adeno-associated viral vector for efficient delivery of two genes to dorsal root ganglion neurons. | Q42252512 | ||
| Intracerebroventricular injection of adeno-associated virus 6 and 9 vectors for cell type-specific transgene expression in the spinal cord | Q42257686 | ||
| Construction and analysis of compact muscle-specific promoters for AAV vectors | Q42437569 | ||
| Differential transgene expression in brain cells in vivo and in vitro from AAV-2 vectors with small transcriptional control units. | Q43764350 | ||
| Efficient gene transduction of neurons by lentivirus with enhanced neuron-specific promoters. | Q45264367 | ||
| Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector | Q45598603 | ||
| Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes. | Q45735544 | ||
| Neuron-specific transduction in the rat septohippocampal or nigrostriatal pathway by recombinant adeno-associated virus vectors | Q45756988 | ||
| Quantitative analysis of the packaging capacity of recombinant adeno-associated virus | Q45766162 | ||
| Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector | Q45769447 | ||
| Promoter-specific lentivectors for long-term, cardiac-directed therapy of Fabry disease | Q45863614 | ||
| Influence of promoter and WHV post-transcriptional regulatory element on AAV-mediated transgene expression in the rat brain | Q45869654 | ||
| Human CMV immediate-early enhancer: a useful tool to enhance cell-type-specific expression from lentiviral vectors | Q45872906 | ||
| Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state | Q45872960 | ||
| CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in mice | Q45875988 | ||
| Phenotypic correction and long-term expression of factor VIII in hemophilic mice by immunotolerization and nonviral gene transfer using the Sleeping Beauty transposon system | Q45879828 | ||
| A comparative analysis of constitutive and cell-specific promoters in the adult mouse hippocampus using lentivirus vector-mediated gene transfer | Q45880149 | ||
| Human thyroxine binding globulin (TBG) promoter directs efficient and sustaining transgene expression in liver-specific pattern | Q45880673 | ||
| Optimization of plasmid vectors for high-level expression in lung epithelial cells | Q45881255 | ||
| GFAP promoter directs astrocyte-specific expression in transgenic mice | Q48163933 | ||
| A very strong enhancer is located upstream of an immediate early gene of human cytomegalovirus | Q48378635 | ||
| Coding and potential regulatory sequences of a cluster of chorion genes in Drosophila melanogaster | Q48383860 | ||
| Dose and promoter effects of adeno-associated viral vector for green fluorescent protein expression in the rat brain | Q48563498 | ||
| Cytokine stimulation and the choice of promoter are critical factors for the efficient transduction of mouse T cells with HIV-1 vectors. | Q50572029 | ||
| Improved cardiac gene transfer by transcriptional and transductional targeting of adeno-associated viral vectors. | Q51278877 | ||
| Effect of promoters and enhancers on expression, transgene DNA persistence, and hepatotoxicity after adenoviral gene transfer of human apolipoprotein A-I | Q64378906 | ||
| In vitro polyadenylation is stimulated by the presence of an upstream intron | Q67676618 | ||
| Definition of an efficient synthetic poly(A) site | Q69373727 | ||
| GFAP promoter elements required for region-specific and astrocyte-specific expression | Q80607573 | ||
| P433 | issue | 102 | |
| P921 | main subject | gene therapy | Q213901 |
| P304 | page(s) | 49-57 | |
| P577 | publication date | 2015-01-01 | |
| P1433 | published in | Discovery Medicine | Q5281940 |
| P1476 | title | Viral expression cassette elements to enhance transgene target specificity and expression in gene therapy | |
| P478 | volume | 19 |
| Q57839812 | A Library-Based Screening Strategy for the Identification of DARPins as Ligands for Receptor-Targeted AAV and Lentiviral Vectors |
| Q37313599 | A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome |
| Q39409371 | Adeno-Associated Virus (AAV) as a Vector for Gene Therapy. |
| Q55339450 | Autism spectrum disorder: prospects for treatment using gene therapy. |
| Q92487836 | Challenges in treating Pompe disease: an industry perspective |
| Q38659647 | Characterisation of a stably integrated expression system for exogenous protein expression in DT40. |
| Q36792325 | Enhancing Transgene Expression from Recombinant AAV8 Vectors in Different Tissues Using Woodchuck Hepatitis Virus Post-Transcriptional Regulatory Element |
| Q92155542 | Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion |
| Q59349001 | Gene Therapy Strategies to Restore ER Proteostasis in Disease |
| Q39144904 | Genome Editing for the Study of Cardiovascular Diseases |
| Q26823895 | Lipid Nanoparticles for Ocular Gene Delivery |
| Q90549130 | Mitigating off-target effects in CRISPR/Cas9-mediated in vivo gene editing |
| Q37698337 | Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease. |
| Q55039934 | Progress in Gene Therapy to Prevent Retinal Ganglion Cell Loss in Glaucoma and Leber's Hereditary Optic Neuropathy. |
| Q54206545 | Recent progress and considerations for AAV gene therapies targeting the central nervous system. |
| Q92482655 | Restoration of insulin receptor improves diabetic phenotype in T2DM mice |
| Q36035457 | STAT1 modification improves therapeutic effects of interferons on lung cancer cells |
| Q58806285 | Sertoli Cells Engineered to Express Insulin to Lower Blood Glucose in Diabetic Mice |
| Q94528057 | Small Alphaherpesvirus Latency-Associated Promoters Drive Efficient and Long-Term Transgene Expression in the CNS |
| Q37127728 | Strategies for targeting primate neural circuits with viral vectors |
| Q58726929 | Surface-Engineered Lentiviral Vectors for Selective Gene Transfer into Subtypes of Lymphocytes |
| Q64263017 | Tumor-Specific Delivery of Immune Checkpoint Inhibitors by Engineered AAV Vectors |
| Q30376584 | Viral-mediated Ntf3 overexpression disrupts innervation and hearing in nondeafened guinea pig cochleae |
| Q89878841 | rAAV-Mediated Cochlear Gene Therapy: Prospects and Challenges for Clinical Application |
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