All Stories
- Nicholas Muzyczka, PhD [1947–2023]
Article
- Development of an AAV DNA-based synthetic vector for the potential gene therapy of hemophilia in children
Article
- Gene Therapy for Hemophilia A
Article
- Age‐stratified adeno‐associated virus serotype 3 neutralizing and total antibody prevalence in hemophilia A patients from India
Article
- Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette
Article
- Reply to “D” matters in recombinant AAV packaging
- Prevalence of Adeno-Associated Virus 3 Capsid Binding and Neutralizing Antibodies in Healthy and Hemophilia B Individuals from India
Article
- Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse
- When the Capsid Meets the Promoter: A New Insight into Transgene Expression from Adeno-Associated Virus Vectors
- Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B
Article
- Role of Essential Metal Ions in AAV Vector-Mediated Transduction
Article
- AAV Vectors: Are They Safe?
Article
- Enhanced Transduction of Human Hematopoietic Stem Cells by AAV6 Vectors: Implications in Gene Therapy and Genome Editing
- Site-Directed Mutagenesis Improves the Transduction Efficiency of Capsid Library-Derived Recombinant AAV Vectors
- A Tribute to Barrie J. Carter
- Adeno-Associated Virus D-Sequence-Mediated Suppression of Expression of a Human Major Histocompatibility Class II Gene: Implications in the Development of Adeno-Associated Virus Vectors for Modulating Humoral Immune Response
- AAV3-miRNA vectors for growth suppression of human hepatocellular carcinoma cells in vitro and human liver tumors in a murine xenograft model in vivo
Article
- Next Generation of Adeno-Associated Virus Vectors for Gene Therapy for Human Liver Diseases
- Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors
- XVIIth International Parvovirus Workshop
- Chinese Medicine Protein and Peptide in Gene and Cell Therapy
- The hepatocyte-specific HNF4α/miR-122 pathway contributes to iron overload–mediated hepatic inflammation
- Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8+ T cells
- Authors' Response to Jesse D. Riordan, Hum Gene Ther 2017;28:375–376; DOI: 10.1089/hum.2017.045
- Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models
- AAV Infection: Protection from Cancer
- Efficient Gene Delivery and Expression in Pancreas and Pancreatic Tumors by Capsid-Optimized AAV8 Vectors
- In vivo tissue-tropism of adeno-associated viral vectors
- High-Efficiency Transduction of Primary Human Hematopoietic Stem/Progenitor Cells by AAV6 Vectors: Strategies for Overcoming Donor-Variation and Implications in Genome Editing
- Development of Optimized AAV Serotype Vectors for High-Efficiency Transduction at Further Reduced Doses
- Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid
- A Tribute to George Stamatoyannopoulos
- Adeno-Associated Virus: The Naturally Occurring Virus Versus the Recombinant Vector
- Microglia-specific targeting by novel capsid-modified AAV6 vectors
- Strategies to generate high-titer, high-potency recombinant AAV3 serotype vectors
- Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?
- Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors
- Site-Directed Mutagenesis of Surface-Exposed Lysine Residues Leads to Improved Transduction by AAV2, But Not AAV8, Vectors in Murine Hepatocytes In Vivo
- Prevalence of neutralizing antibodies against liver-tropic adeno-associated virus serotype vectors in 100 healthy Chinese and its potential relation to body constitutions
- Productive life cycle of adeno-associated virus serotype 2 in the complete absence of a conventional polyadenylation signal
- SelectiveIn VivoTargeting of Human Liver Tumors by Optimized AAV3 Vectors in a Murine Xenograft Model
- Cytotoxic genes from traditional Chinese medicine inhibit tumor growth both in vitro and in vivo
- Enhanced Transgene Expression from Recombinant Single-Stranded D-Sequence-Substituted Adeno-Associated Virus Vectors in Human Cell LinesIn Vitroand in Murine HepatocytesIn Vivo
- Efficient lysis of epithelial ovarian cancer cells by MAGE-A3-induced cytotoxic T lymphocytes using rAAV-6 capsid mutant vector
- Reprogramming Adipose Tissue-Derived Mesenchymal Stem Cells into Pluripotent Stem Cells by a Mutant Adeno-Associated Viral Vector
- Pristimerin enhances recombinant adeno-associated virus vector-mediated transgene expression in human cell lines in vitro and murine hepatocytes in vivo
- Lignin Nanotubes As Vehicles for Gene Delivery into Human Cells
- Molecular cloning, overexpression, and an efficient one-step purification of α5β1 integrin
- Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo
- Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells
- Optimization of the Capsid of Recombinant Adeno-Associated Virus 2 (AAV2) Vectors: The Final Threshold?
- High-Efficiency Transduction of Primary Human Hematopoietic Stem Cells and Erythroid Lineage-Restricted Expression by Optimized AAV6 Serotype Vectors In Vitro and in a Murine Xenograft Model In Vivo
- Limitations of Encapsidation of Recombinant Self-Complementary Adeno-Associated Viral Genomes in Different Serotype Capsids and Their Quantitation
- High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors
- A Simplified Immune Suppression Scheme Leads to Persistent Micro-dystrophin Expression in Duchenne Muscular Dystrophy Dogs
- Role of Molecular Genetics in Hemophilia: From Diagnosis to Therapy
- Recombinant Adeno-Associated Virus-Mediated Gene Transfer for the Potential Therapy of Adenosine Deaminase-Deficient Severe Combined Immune Deficiency
- Development of optimized AAV3 serotype vectors: mechanism of high-efficiency transduction of human liver cancer cells
- A Simple Method to Increase the Transduction Efficiency of Single-Stranded Adeno-Associated Virus VectorsIn VitroandIn Vivo
- Cellular fusion for gene delivery to SCA1 affected Purkinje neurons
- High-Efficiency Transduction of Liver Cancer Cells by Recombinant Adeno-Associated Virus Serotype 3 Vectors
- Novel Properties of Tyrosine-mutant AAV2 Vectors in the Mouse Retina
- Innate Immune Responses to AAV Vectors
- High-efficiency Transduction and Correction of Murine Hemophilia B Using AAV2 Vectors Devoid of Multiple Surface-exposed Tyrosines
- Human Hepatocyte Growth Factor Receptor Is a Cellular Coreceptor for Adeno-Associated Virus Serotype 3
- High-Efficiency Transduction of Fibroblasts and Mesenchymal Stem Cells by Tyrosine-Mutant AAV2 Vectors for Their Potential Use in Cellular Therapy
- Adeno-Associated Virus Serotype 6 Capsid Tyrosine-to-Phenylalanine Mutations Improve Gene Transfer to Skeletal Muscle
- An animal model of PDH deficiency using AAV8-siRNA vector-mediated knockdown of pyruvate dehydrogenase E1α
- Enhanced Long-Term Transduction and Multilineage Engraftment of Human Hematopoietic Stem Cells Transduced with Tyrosine-Modified Recombinant Adeno-Associated Virus Serotype 2
- Optimized Adeno-Associated Virus (AAV)–Protein Phosphatase-5 Helper Viruses for Efficient Liver Transduction by Single-Stranded AAV Vectors: Therapeutic Expression of Factor IX at Reduced Vector Doses
- AAV3-mediated transfer and expression of the pyruvate dehydrogenase E1 alpha subunit gene causes metabolic remodeling and apoptosis of human liver cancer cells
- Antiviral activity of copper complexes of isoniazid against RNA tumor viruses
- Calpeptin Increases the Activity of Upstream Stimulatory Factor and Induces High Level Globin Gene Expression in Erythroid Cells
- High-efficiency Transduction of the Mouse Retina by Tyrosine-mutant AAV Serotype Vectors
- Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression
- Adeno‐associated virus‐mediated gene transfer
- Correction for Zhong et al. , Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
- Working group report: the roles of glycans in hemostasis, inflammation and vascular biology
- Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
- Strategies for improving the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo
- A combined therapeutic approach for pyruvate dehydrogenase deficiency using self-complementary adeno-associated virus serotype-specific vectors and dichloroacetate
- Optimization of Recombinant Adeno-Associated Viral Vectors for Humanβ-Globin Gene Transfer and Transgene Expression
- Recombinant Self-Complementary Adeno-Associated Virus Serotype Vector-Mediated Hematopoietic Stem Cell Transduction and Lineage-Restricted, Long-Term Transgene Expression in a Murine Serial Bone Marrow Transplantation Model
- Stable Integration of Recombinant Adeno-Associated Virus Vector Genomes After Transduction of Murine Hematopoietic Stem Cells
- Single-polarity Recombinant Adeno-associated Virus 2 Vector-mediated Transgene Expression In Vitro and In Vivo: Mechanism of Transduction
- Pyruvate Dehydrogenase Complex Deficiency Caused by Ubiquitination and Proteasome-mediated Degradation of the E1β Subunit
- A Dual Role of EGFR Protein Tyrosine Kinase Signaling in Ubiquitination of AAV2 Capsids and Viral Second-strand DNA Synthesis
- Down-regulation of expression of rat pyruvate dehydrogenase E1α gene by self-complementary adeno-associated virus-mediated small interfering RNA delivery
- Self-Complementary Recombinant Adeno-Associated Viral Vectors: Packaging Capacity And The Role of Rep Proteins in Vector Purity
- Adeno-Associated Virus-Mediated Gene Transfer in Hematopoietic Stem/Progenitor Cells as a Therapeutic Tool
- Adeno-associated virus 2-mediated gene transfer: role of a cellular serine/threonine protein phosphatase in augmenting transduction efficiency
- FK506-Binding Protein 52 Is Essential to Uterine Reproductive Physiology Controlled by the Progesterone Receptor A Isoform
- Role of cellular FKBP52 protein in intracellular trafficking of recombinant adeno-associated virus 2 vectors
- Role of Integrin Cross-Regulation in Parvovirus B19 Targeting
- Evaluation of Primitive Murine Hematopoietic Stem and Progenitor Cell Transduction In Vitro and In Vivo by Recombinant Adeno-Associated Virus Vector Serotypes 1 Through 5
- Hematopoietic Stem Cell Transduction by Recombinant Adeno-Associated Virus Vectors: Problems and Solutions
- Heat-shock Treatment-mediated Increase in Transduction by Recombinant Adeno-associated Virus 2 Vectors Is Independent of the Cellular Heat-shock Protein 90
- Recombinant human parvovirus B19 vectors
- Adeno-Associated Virus Type 2-Mediated Gene Transfer: Role of Cellular FKBP52 Protein in Transgene Expression
- Infection of Purified Nuclei by Adeno-associated Virus 2
- Adeno-associated Virus 2-Mediated Transduction and Erythroid Lineage-Restricted Long-Term Expression of the Human β-Globin Gene in Hematopoietic Cells from Homozygous β-Thalassemic Mice
- Adeno-Associated Virus Type 2-Mediated Gene Transfer: Altered Endocytic Processing Enhances Transduction Efficiency in Murine Fibroblasts
- Recombinant Human Parvovirus B19 Vectors: Erythrocyte P Antigen Is Necessary but Not Sufficient for Successful Transduction of Human Hematopoietic Cells
- Gene Therapy with Viral Vectors: The Hope, the Problems, and the Solution
- Impaired Intracellular Trafficking of Adeno-Associated Virus Type 2 Vectors Limits Efficient Transduction of Murine Fibroblasts
- Erythroviruses as Gene Transfer Vehicles
- Adeno-Associated Virus 2-Mediated Transduction and Erythroid Lineage-Restricted Expression from Parvovirus B19p6 Promoter in Primary Human Hematopoietic Progenitor Cells
- Adeno-associated virus 2 co-receptors?
- Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2
- Adeno-Associated Virus Type 2-Mediated Gene Transfer: Role of Epidermal Growth Factor Receptor Protein Tyrosine Kinase in Transgene Expression
- Characterization of Wild-Type Adeno-Associated Virus Type 2-Like Particles Generated during Recombinant Viral Vector Production and Strategies for Their Elimination
- Recombinant Human Parvovirus B19 Vectors: Erythroid Cell-Specific Delivery and Expression of Transduced Genes
- Rescue and Autonomous Replication of Adeno-Associated Virus Type 2 Genomes Containing Rep-Binding Site Mutations in the Viral p5 Promoter
- Adeno-Associated Virus Type 2-Mediated Gene Transfer: Correlation of Tyrosine Phosphorylation of the Cellular Single-Stranded D Sequence-Binding Protein with Transgene Expression in Human Cells In Vitro and Murine Tissues In Vivo
- Adeno-associated virus type 2-mediated transduction in primary human bone marrow-derived CD34+ hematopoietic progenitor cells: donor variation and correlation of transgene expression with cellular differentiation
- Encapsidation of adeno-associated virus type 2 Rep proteins in wild-type and recombinant progeny virions: Rep-mediated growth inhibition of primary human cells
- Role of tyrosine phosphorylation of a cellular protein in adeno-associated virus 2-mediated transgene expression
- Quantitative DNA slot blot analysis: inhibition of DNA binding to membranes by magnesium ions
- Adeno-associated virus type 2-mediated transfer of ecotropic retrovirus receptor cDNA allows ecotropic retroviral transduction of established and primary human cells
- Adeno-associated virus type 2 DNA replication in vivo: mutation analyses of the D sequence in viral inverted terminal repeats
- Adeno-associated virus type 2-mediated transduction of murine hematopoietic cells with long-term repopulating ability and sustained expression of a human globin gene in vivo
- Lack of Site-Specific Integration of the Recombinant Adeno-Associated Virus 2 Genomes in Human Cells
- A novel terminal resolution-like site in the adeno-associated virus type 2 genome
- Adeno-associated virus 2-mediated gene transfer in vivo: organ-tropism and expression of transduced sequences in mice
- Evaluation of recombinant adeno-associated virus as a gene transfer vector for the retina
- Differential expression in human cells from the p6 promoter of human parvovirus B19 following plasmid transfection and recombinant adeno-associated virus 2 (AAV) infection: human megakaryocytic leukaemia cells are non-permissive for AAV infection
- Rescue and replication of adeno-associated virus type 2 as well as vector DNA sequences from recombinant plasmids containing deletions in the viral inverted terminal repeats: selective encapsidation of viral genomes in progeny virions
- Adeno-associated Virus 2-Mediated Transduction and Erythroid Lineage-Specific Expression in Human Hematopoietic Progenitor Cells
- Parvovirus B19 promoter at map unit 6 confers autonomous replication competence and erythroid specificity to adeno-associated virus 2 in primary human hematopoietic progenitor cells.
- Cord Blood Transplantation and the Potential for Gene Therapy
- Transcriptional transactivation of parvovirus B19 promoters in nonpermissive human cells by adenovirus type 2
- Transduction of folate receptor cDNA into cervical carcinoma cells using recombinant adeno-associated virions delays cell proliferation in vitro and in vivo.
- Rescue and Replication Signals of the Adeno-associated Virus 2 Genome
- Adeno-associated virus 2-mediated high efficiency gene transfer into immature and mature subsets of hematopoietic progenitor cells in human umbilical cord blood.
- Molecular analysis of simple variant translocations in acute promyelocytic leukemia
- Suppression of human alpha-globin gene expression mediated by the recombinant adeno-associated virus 2-based antisense vectors.
- Biochemical and antitumor activity of trimidox, a new inhibitor of ribonucleotide reductase
- Versatile adeno-associated virus 2-based vectors for constructing recombinant virions
- Successful replication of parvovirus B19 in the human megakaryocytic leukemia cell line MB-02
- Cloning and integration of DNA fragments in human cells via the inverted terminal repeats of the adeno-associated virus 2 genome
- Parvovirus B19 replication in human umbilical cord blood cells
- Rescue of the Adeno-Associated Virus 2 Genome Correlates with Alterations in DNA-Modifying Enzymes in Human Cells
- Parvovirus B19-induced perturbation of human megakaryocytopoiesis in vitro
- Absence of reverse transcriptase activity in human diploid fibroblasts
- Construction of a recombinant human parvovirus B19: adeno-associated virus 2 (AAV) DNA inverted terminal repeats are functional in an AAV-B19 hybrid virus.
- Identification of nuclear proteins that specifically interact with adeno-associated virus type 2 inverted terminal repeat hairpin DNA
- Augmented nuclease activity during cellular senescence in vitro
- Rescue and Replication of the Adeno-Associated Virus 2 Genome in Mortal and Immortal Human Cells
- KRAS2 oncogene overexpression in myelodysplastic syndrome with translocation 5;12
- Replication of B19 parvovirus in highly enriched hematopoietic progenitor cells from normal human bone marrow
- A variant t(X;15)(p11;q22) translocation in acute promyelocytic leukemia
- Replication of the Adeno-Associated Virus DNA Termini in vitro
- Human alphoid family of tandemly repeated DNA
- Changes in Genetic Organization and Expression in Aging Cells
- Autocrine regulation of growth: II. Glucocorticoids inhibit transcription of c-sis oncogene-specific RNA transcripts
- The Genetics of Adeno-Associated Virus
- Rescue of adeno-associated virus from recombinant plasmids: Gene correction within the terminal repeats of AAV
- Nucleotide sequence and organization of the adeno-associated virus 2 genome
- Observations on the phosphonoformic acid inhibition of RNA dependent DNA polymerases
- Biochemistry of terminal deoxynucleotidyltransferase: identification, characterization, requirements, and active-site involvement in the catalysis of associated pyrophosphate exchange and pyrophosphorolytic activity
- Reverse transcriptase-associated RNase H. Part IV. Pyrophosphate does not inhibit RNase H activity of AMV DNA polymerase
- Molecular mechanism for the specific inhibition of reverse transcriptase of rous sarcoma virus by the copper complexes of isonicotinic acid hydrazide