Genetic Fate of Recombinant Adeno-Associated Virus Vector Genomes in Muscle

No abstract is available for this paper.

• Publication year

  2003

• Venue

  Journal of Virology

• Publication date

  2003-03-15

• Fields of study

  Biology, Medicine

• Identifiers
  DOI 10.1128/JVI.77.6.3495-3504.2003PMID 12610125PMCID PMC149530
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

• Chromosomal effects of adeno-associated virus vector integration

  2002cited by this paper
• Effect Of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle

  2001cited by this paper
• Molecular Cloning: A Laboratory Manual

  2001cited by this paper
• A quantitative assay for HIV DNA integration in vivo

  2001cited by this paper
• Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors

  2001cited by this paper
• Extrachromosomal Recombinant Adeno-Associated Virus Vector Genomes Are Primarily Responsible for Stable Liver Transduction In Vivo

  2001cited by this paper
• Gene transfer as an approach to treating hemophilia.

  2001cited by this paper
• Plasmid DNA Vaccines: Investigation of Integration into Host Cellular DNA following Intramuscular Injection in Mice

  2001cited by this paper
• Adeno-associated virus vectors for gene therapy: more pros than cons?

  2000cited by this paper
• Kinetics of Recombinant Adeno-Associated Virus-Mediated Gene Transfer

  2000cited by this paper
• Recruitment of Single-Stranded Recombinant Adeno-Associated Virus Vector Genomes and Intermolecular Recombination Are Responsible for Stable Transduction of Liver In Vivo

  2000cited by this paper
• Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector

  2000cited by this paper
• Evolutionary History of B1 Retroposons in the Genus Mus

  2000cited by this paper
• In vivo selection of hepatocytes transduced with adeno-associated viral vectors.

  2000cited by this paper
• Latent Adeno-Associated Virus Infection Elicits Humoral but Not Cell-Mediated Immune Responses in a Nonhuman Primate Model

  1999cited by this paper
• Gene transfer into the CNS using recombinant adeno-associated virus: analysis of vector DNA forms resulting in sustained expression.

  1999influential reference
• Structure of Adeno-Associated Virus Vector DNA following Transduction of the Skeletal Muscle

  1999cited by this paper
• Immune responses to adenovirus and adeno-associated virus in humans

  1999cited by this paper
• Concatamerization of Adeno-Associated Virus Circular Genomes Occurs through Intermolecular Recombination

  1999cited by this paper
• Safety and Biological Efficacy of an Adeno‐Associated Virus Vector–Cystic Fibrosis Transmembrane Regulator (AAV‐CFTR) in the Cystic Fibrosis Maxillary Sinus

  1999cited by this paper
• Plasmid DNA Malaria Vaccine: The Potential for Genomic Integration after Intramuscular Injection

  1999cited by this paper
• Highly Purified Recombinant Adeno-Associated Virus Vectors Are Biologically Active and Free of Detectable Helper and Wild-Type Viruses

  1999influential reference
• Isolation of Recombinant Adeno-Associated Virus Vector-Cellular DNA Junctions from Mouse Liver

  1999cited by this paper
• Frequency and Stability of Chromosomal Integration of Adenovirus Vectors

  1999cited by this paper
• The kinetics of rAAV integration in the liver

  1998cited by this paper
• Circular Intermediates of Recombinant Adeno-Associated Virus Have Defined Structural Characteristics Responsible for Long-Term Episomal Persistence in Muscle Tissue

  1998cited by this paper
• Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus.

  1998cited by this paper
• Adeno-Associated Virus Vector-Mediated Transgene Integration into Neurons and Other Nondividing Cell Targets

  1998cited by this paper
• Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors.

  1997cited by this paper
• Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line

  1997cited by this paper
• Adeno-associated virus vector integration junctions

  1997cited by this paper
• Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro

  1997cited by this paper
• Recombinant Adeno-Associated Viral Vectors Mediate Long-Term Transgene Expression in Muscle

  1997cited by this paper
• Recombinant adeno-associated virus for muscle directed gene therapy

  1997influential reference
• Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

  1997cited by this paper
• Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus.

  1997cited by this paper
• In vivo model of adeno-associated virus vector persistence and rescue

  1996cited by this paper
• Recombinant adeno-associated virus (AAV-CFTR) vectors do not integrate in a site-specific fashion in an immortalized epithelial cell line.

  1996cited by this paper
• Recombinant adeno-associated virus-mediated high-efficiency, transient expression of the murine cationic amino acid transporter (ecotropic retroviral receptor) permits stable transduction of human HeLa cells by ecotropic retroviral vectors

  1996cited by this paper
• Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector

  1996cited by this paper
• Cell Lines for the Production of Recombinant Adeno-Associated Virus

  1995cited by this paper
• Recombinant junctions formed by site-specific integration of adeno-associated virus into an episome

  1995cited by this paper
• Genotoxicity studies with chloroethane.

  1994cited by this paper
• International Commission for Protection Against Environmental Mutagens and Carcinogens. Working paper no. 3. Somatic mutant frequency, mutation rates and mutational spectra in the human population in vivo.

  1994cited by this paper
• Direct gene transfer into muscle.

  1994cited by this paper
• Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA.

  1994cited by this paper
• Plasmid integration, amplification and cytogenetics in CHO cells: questions and comments.

  1994cited by this paper
• The immunobiology of muscle.

  1994cited by this paper
• Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector.

  1993cited by this paper
• Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector.

  1992cited by this paper
• Manipulation of adenovirus vectors.

  1991cited by this paper
• Targeted integration of adeno‐associated virus (AAV) into human chromosome 19.

  1991cited by this paper
• Overview of biological effects of addition of DNA molecules to cells.

  1990cited by this paper
• Site-specific integration by adeno-associated virus.

  1990cited by this paper
• Overview of biological effects of addition of DNA molecules to cells

  1990cited by this paper
• Site-specific integration by adeno-associated virus.

  1990cited by this paper
• Cationic liposome-mediated transfection

  1989cited by this paper
• Cationic liposome mediated transfection.

  1989cited by this paper
• Adeno-associated virus general transduction vectors: analysis of proviral structures

  1988cited by this paper
• Adeno-associated virus: a vector system for efficient introduction and integration of DNA into a variety of mammalian cell types

  1988cited by this paper
• Oligonucleotide probes for the screening of recombinant DNA libraries.

  1987cited by this paper
• Guide to molecular cloning techniques.

  1987cited by this paper
• Oligonucleotide probes for the screening of recombinant DNA libraries

  1987cited by this paper
• Adeno-associated virus vector for high-frequency integration, expression, and rescue of genes in mammalian cells

  1985cited by this paper
• Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells.

  1984cited by this paper
• Perspectives in mutation epidemiology: 3. Contribution of chromosome abnormalities to human morbidity and mortality and some comments upon surveillance of chromosome mutation rates

  1983cited by this paper
• International Commission for Protection Against Environmental Mutagens and Carcinogens.

  1979cited by this paper
• A colony bank containing synthetic CoI EI hybrid plasmids representative of the entire E. coli genome

  1976cited by this paper

• Nanovaccine enables complement system inhibition and high-dose AAV re-administration

  2025cites this paper
• AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects

  2025cites this paper
• AAV Genome Topology Decides ITR Secondary Structure

  2025cites this paper
• Adeno-Associated Virus-Induced Neurotoxicity is Prevented by CpG Depletion

  2025cites this paper
• The Hepatic Odyssey of AAV: From Gene Delivery to Long-Term Outcomes.

  2025cites this paper
• Durable lymphocyte subset elimination upon a single dose of AAV-delivered depletion antibody dissects immune control of chronic viral infection.

  2024cites this paper
• Adeno-Associated Viral Vector Integration: Implications for Long-Term Efficacy and Safety.

  2024cites this paper
• Prenatal AAV9-GFP administration in fetal lambs results in transduction of female germ cells and maternal exposure to virus

  2024cites this paper
• Advances in Bone-Targeting Drug Delivery: Emerging Strategies Using Adeno-Associated Virus

  2024cites this paper
• Natural Adeno-Associated Virus Serotypes and Engineered Adeno-Associated Virus Capsid Variants: Tropism Differences and Mechanistic Insights

  2024cites this paper
• AAV-DJ is superior to AAV9 for targeting brain and spinal cord, and de-targeting liver across multiple delivery routes in mice

  2024cites this paper
• Gene Therapy for Hemophilia B: Achievements, Open Issues, and Perspectives

  2024cites this paper
• Biological control of Pseudomonas aeruginosa (ATCC-27853) by isolated lytic bacteriophage

  2024cites this paper
• Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog

  2023cites this paper
• Duchenne muscular dystrophy: disease mechanism and therapeutic strategies

  2023cites this paper
• SCN1A as a therapeutic target for Dravet syndrome

  2023cites this paper
• Recent advances in therapeutic CRISPR-Cas9 genome editing: mechanisms and applications

  2023cites this paper
• Whole-Body Disposition and Physiologically Based Pharmacokinetic Modeling of Adeno-Associated Viruses and the Transgene Product.

  2023cites this paper
• Random Integration Analysis of Recombinant Adeno-Associated Virus 6 Packaged in Sf9 Insect Cells

  2023cites this paper
• Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence

  2022cites this paper
• Mitochondrial DNA replication and repair defects: Clinical phenotypes and therapeutic interventions.

  2022cites this paper
• Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy

  2022cites this paper
• Immune profiling of adeno-associated virus response identifies B cell-specific targets that enable vector re-administration in mice

  2022cites this paper
• Red Blood Cell Anchoring Enables Targeted Transduction and Re‐Administration of AAV‐Mediated Gene Therapy

  2022cites this paper
• Vasculogenic gene therapy: No role for revitalization of structural bone allografts

  2022cites this paper
• A review of the underlying genetics and emerging therapies for canine cardiomyopathies.

  2021cites this paper
• Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?

  2021cites this paper
• Barth syndrome: cardiolipin, cellular pathophysiology, management, and novel therapeutic targets

  2021cites this paper
• TAZ encodes tafazzin, a transacylase essential for cardiolipin formation and central to the etiology of Barth syndrome.

  2020cites this paper
• Toxicological Aspects in the Regulation of Gene Therapy Medicinal Products

  2020cites this paper
• Mouse γ-Synuclein Promoter-Mediated Gene Expression and Editing in Mammalian Retinal Ganglion Cells

  2020cites this paper
• Moving towards clinical trials for mitochondrial diseases

  2020cites this paper
• Barth syndrome cardiomyopathy: targeting the mitochondria with elamipretide

  2020cites this paper
• Vascular Tumors Result from Adeno-Associated Virus-9 Angiogenic Gene Therapy of Bone Allografts

  2020cites this paper
• Parkinson’s Disease: An Overview of Various Treatments

  2020cites this paper
• R-loop-forming Sequences Analysis in Thousands of Viral Genomes Identify A New Common Element in Herpesviruses

  2020cites this paper
• My Pathway to Adeno-Associated Virus and Adeno-Associated Virus Gene Therapy: A Personal Perspective

  2020cites this paper
• Downregulation of α-Synuclein Protein Levels by an Intracellular Single-Chain Antibody

  2020cites this paper
• Chemogenetics a Robust Approach to Pharmacology and Gene Therapy.

  2020cites this paper
• The Unusual Properties of the AAV Inverted Terminal Repeat

  2020cites this paper
• Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays

  2020cites this paper
• Cervical Gene Delivery of the Antimicrobial Peptide, Human β-Defensin (HBD)-3, in a Mouse Model of Ascending Infection-Related Preterm Birth

  2019cites this paper
• Vector uncoating limits adeno-associated viral vector-mediated transduction of human dendritic cells and vector immunogenicity

  2019cites this paper
• An investigation into the role of mesoaccumbal GABAA receptor α2 subunit in mediating cocaine-facilitated conditioned behaviours using the RNA interference system

  2019cites this paper
• Adeno-associated virus (AAV)–mediated gene therapy is under investigation as a thera- peutic option for persons with hemophilia A. Efficacy and safety data include 3 years of follow-up after a single administration of AAV5-hFVIII-SQ

  2019cites this paper
• AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.

  2019cites this paper
• Increased mtDNA Abundance and Improved Function in Human Barth Syndrome Patient Fibroblasts Following AAV-TAZ Gene Delivery

  2019cites this paper
• Identification of neuron-type specific promoters in monkey genome and their functional validation in mice.

  2019cites this paper
• AAV-Mediated TAZ Gene Replacement Restores Mitochondrial and Cardioskeletal Function in Barth Syndrome

  2019cites this paper
• Novel AAV-mediated gene delivery system corrects CFTR function in pigs.

  2019cites this paper
• High levels of AAV vector integration into CRISPR-induced DNA breaks

  2019cites this paper
• T5 Exonuclease Hydrolysis of Hepatitis B Virus Replicative Intermediates Allows Reliable Quantification and Fast Drug Efficacy Testing of Covalently Closed Circular DNA by PCR

  2018cites this paper
• Personalized gene and cell therapy for Duchenne Muscular Dystrophy.

  2018cites this paper
• Novel Approaches for Pluripotent Reprogramming

  2018cites this paper
• Cerebrospinal Fluid-Directed rAAV9-rsATP7A Plus Subcutaneous Copper Histidinate Advance Survival and Outcomes in a Menkes Disease Mouse Model

  2018cites this paper
• 7 Gene Therapy in Cardiovascular Disease

  2018cites this paper
• Adeno-associated virus gene delivery of broadly neutralizing antibodies as prevention and therapy against HIV-1

  2018cites this paper
• Development of Methodology and Study Protocol: Safety and Efficacy of a Single Subretinal Injection of rAAV.hCNGA3 in Patients with CNGA3-Linked Achromatopsia Investigated in an Exploratory Dose-Escalation Trial

  2018cites this paper
• The Role of the Human Bocavirus (HBoV) in Respiratory Infections

  2018cites this paper
• The use of cervical gene therapy for the prevention of infection-related preterm birth

  2017cites this paper
• AAV Infection: Protection from Cancer

  2017cites this paper
• Développement d'une stratégie thérapeutique pour la dystrophie facio-scapulo-humérale.

  2017cites this paper
• A Little Help From the Follicles: Understanding the Germinal Center Response to Human Immunodeficiency Virus 1 Infection and Prophylactic Vaccines

  2017cites this paper
• Strategien zur Inhibierung der HIV-Reaktiverung in latent infizierten Astrozyten

  2017cites this paper
• Modern biotechnology-based therapeutic approaches against HIV infection.

  2017cites this paper
• Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models

  2017cites this paper
• Engineered Expression of Broadly Neutralizing Antibodies Against Human Immunodeficiency Virus.

  2017cites this paper
• Progress toward Gene Therapy for Duchenne Muscular Dystrophy.

  2017cites this paper
• Gene therapy for hypertension

  2017cites this paper
• Engineering broadly neutralizing antibodies for HIV prevention and therapy.

  2016cites this paper
• Delivery methods for site-specific nucleases: Achieving the full potential of therapeutic gene editing.

  2016cites this paper
• Promise and problems associated with the use of recombinant AAV for the delivery of anti-HIV antibodies

  2016cites this paper
• Anti-Angiogenic Gene Therapy: Basic Science and Challenges for Translation into the Clinic

  2016cites this paper
• Utilizing gene therapy methods to probe the genetic requirements to prevent spinal muscular atrophy.

  2016cites this paper
• Intracellular fate of AAV particles in human Dendritic Cell and impact on Gene Transfer

  2016cites this paper
• Restriction Factors Against Recombinant Adeno-associated Virus Vectormediated Gene Transfer in Dystrophin-deficient Muscles.

  2016cites this paper
• neutralizing antibodies for HIV prevention and therapy ☆ O F

  2016cites this paper
• [Integration of AAV vectors and insertional mutagenesis].

  2016cites this paper
• Respiratory infections of the human bocavirus

  2016cites this paper
• Protein-Anchoring Therapy of Biglycan for Mdx Mouse Model of Duchenne Muscular Dystrophy

  2016influential citation
• Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle

  2016cites this paper
• The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo

  2016cites this paper
• Establishment of two quantitative nested qPCR assays targeting the human EPO transgene

  2016cites this paper
• Use of Adeno‐Associated and Herpes Simplex Viral Vectors for In Vivo Neuronal Expression in Mice

  2015cites this paper
• Attenuation of methylglyoxal-induced peritoneal fibrosis: immunomodulation by interleukin-10

  2015cites this paper
• AAV-Delivered Antibody Mediates Significant Protective Effects against SIVmac239 Challenge in the Absence of Neutralizing Activity

  2015cites this paper
• Recombinant AAV-mediated in vivo long-term expression and antitumour activity of an anti-ganglioside GM3(Neu5Gc) antibody

  2015cites this paper
• Vector-mediated antibody gene transfer for infectious diseases.

  2015cites this paper
• RNA-based therapies for dysferlinopathies

  2015cites this paper
• Preclinical Development of New Therapy for Glycogen Storage Diseases.

  2015cites this paper
• ganglioside GM 3 ( Neu 5 Gc ) antibody

  2015cites this paper
• Surface Remodeling of Lentiviral and Adeno-Associated Viral Vectors via Metabolically Introduced Bioorthogonal Functionalities

  2015cites this paper
• Focusing brain therapeutic interventions in space and time for Parkinson's disease.

  2014cites this paper
• AAV-Mediated Gene Therapy for Research and Therapeutic Purposes.

  2014cites this paper
• Low copy number detection of human bocavirus DNA in bronchoalveolar lavage of asymptomatic adult patients

  2014cites this paper
• Adeno-associated virus delivery of broadly neutralizing antibodies

  2014cites this paper
• Vector-Mediated In Vivo Antibody Expression

  2014cites this paper
• Conserved epitopes in Plasmodium falciparum and influenza A virus as targets for virus-vectored immunization

  2014cites this paper
• A Comparative Analysis of Constitutive Promoters Located in Adeno-Associated Viral Vectors

  2014cites this paper
• Safety profile of recombinant adeno-associated viral vectors: focus on alipogene tiparvovec (Glybera®)

  2014cites this paper