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Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success—A Personal Perspective
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What this work says
Outgoing claims: the papers this work cites, builds on, or argues with.
inferredcites →https://doi.org/10.1002/j.1460-2075.1991.tb04964.xopenalex-snapshot
Targeted integration of adeno‐associated virus (AAV) into human chromosome 19.
inferredcites →https://doi.org/10.1002/jgm.885openalex-snapshot
Immune responses to AAV in a phase I study for Canavan disease
inferredcites →https://doi.org/10.1002/jor.20781openalex-snapshot
Systemic human minidystrophin gene transfer improves functions and life span of dystrophin and dystrophin/utrophin‐deficient mice
inferredcites →https://doi.org/10.1006/mthe.2000.0219openalex-snapshot
Several Log Increase in Therapeutic Transgene Delivery by Distinct Adeno-Associated Viral Serotype Vectors
inferredcites →https://doi.org/10.1006/mthe.2001.0449openalex-snapshot
Sustained and Complete Phenotype Correction of Hemophilia B Mice Following Intramuscular Injection of AAV1 Serotype Vectors
inferredcites →https://doi.org/10.1007/978-1-61779-370-7_3openalex-snapshot
AAV Capsid Structure and Cell Interactions
inferredcites →https://doi.org/10.1016/0006-8993(95)01488-8openalex-snapshot
Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector
inferredcites →https://doi.org/10.1016/0042-6822(80)90071-9openalex-snapshot
Assembly of adeno-associated virus
inferredcites →https://doi.org/10.1016/0092-8674(83)90342-2openalex-snapshot
Rescue of adeno-associated virus from recombinant plasmids: Gene correction within the terminal repeats of AAV
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Cloning of infectious adeno-associated virus genomes in bacterial plasmids
inferredcites →https://doi.org/10.1016/j.ymthe.2004.09.011openalex-snapshot
Adeno-associated virus and the development of adeno-associated virus vectors: a historical perspective
inferredcites →https://doi.org/10.1016/s0003-4975(96)00946-0openalex-snapshot
Long-Term Gene Transfer in Porcine Myocardium After Coronary Infusion of an Adeno-Associated Virus Vector
inferredcites →https://doi.org/10.1038/8470openalex-snapshot
Viral receptors and vector purification: New approaches for generating clinical-grade reagents
inferredcites →https://doi.org/10.1038/gt.2008.68openalex-snapshot
Clinical gene therapy using recombinant adeno-associated virus vectors
inferredcites →https://doi.org/10.1038/gt.2009.101openalex-snapshot
Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints
inferredcites →https://doi.org/10.1038/gt.2011.90openalex-snapshot
Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia
inferredcites →https://doi.org/10.1038/mt.2009.155openalex-snapshot
Generation of Novel AAV Variants by Directed Evolution for Improved CFTR Delivery to Human Ciliated Airway Epithelium
inferredcites →https://doi.org/10.1038/mt.2009.292openalex-snapshot
Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB)
inferredcites →https://doi.org/10.1038/mt.2011.237openalex-snapshot
Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
inferredcites →https://doi.org/10.1038/mt.2011.287openalex-snapshot
The AAV Vector Toolkit: Poised at the Clinical Crossroads
inferredcites →https://doi.org/10.1038/nbt.1599openalex-snapshot
Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle
inferredcites →https://doi.org/10.1038/nbt856openalex-snapshot
Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors
inferredcites →https://doi.org/10.1038/ng1094-148openalex-snapshot
Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain
inferredcites →https://doi.org/10.1038/nm0598-635openalex-snapshot
Fluorescent viral vectors: A new technique for the pharmacological analysis of gene therapy
inferredcites →https://doi.org/10.1038/nm1358openalex-snapshot
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
inferredcites →https://doi.org/10.1038/nprot.2006.207openalex-snapshot
Production and characterization of adeno-associated viral vectors
inferredcites →https://doi.org/10.1038/nrg2988openalex-snapshot
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
inferredcites →https://doi.org/10.1038/sj.gt.3300548openalex-snapshot
Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia
inferredcites →https://doi.org/10.1038/sj.gt.3300650openalex-snapshot
In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector
inferredcites →https://doi.org/10.1038/sj.gt.3300938openalex-snapshot
Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield
inferredcites →https://doi.org/10.1038/sj.gt.3301514openalex-snapshot
Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
inferredcites →https://doi.org/10.1038/sj.gt.3302134openalex-snapshot
Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
inferredcites →https://doi.org/10.1056/nejmoa1000228openalex-snapshot
Dystrophin Immunity in Duchenne's Muscular Dystrophy
inferredcites →https://doi.org/10.1073/pnas.0802866105openalex-snapshot
Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
inferredcites →https://doi.org/10.1073/pnas.162250899openalex-snapshot
The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy
inferredcites →https://doi.org/10.1073/pnas.240335297openalex-snapshot
Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
inferredcites →https://doi.org/10.1073/pnas.55.6.1467openalex-snapshot
Studies of small DNA viruses found in various adenovirus preparations: physical, biological, and immunological characteristics.
inferredcites →https://doi.org/10.1073/pnas.64.3.863openalex-snapshot
EVIDENCE FOR A SINGLE-STRANDED ADENOVIRUS-ASSOCIATED VIRUS GENOME: FORMATION OF A DNA DENSITY HYBRID ON RELEASE OF VIRAL DNA
inferredcites →https://doi.org/10.1073/pnas.70.1.215openalex-snapshot
Self-Complementarity of Terminal Sequences Within Plus or Minus Strands of Adenovirus-Associated Virus DNA
inferredcites →https://doi.org/10.1073/pnas.73.3.742openalex-snapshot
Concatemers of alternating plus and minus strands are intermediates in adenovirus-associated virus DNA synthesis.
inferredcites →https://doi.org/10.1073/pnas.76.11.5567openalex-snapshot
Spliced adenovirus-associated virus RNA.
inferredcites →https://doi.org/10.1073/pnas.78.5.2927openalex-snapshot
Eukaryotic translational control: adeno-associated virus protein synthesis is affected by a mutation in the adenovirus DNA-binding protein.
inferredcites →https://doi.org/10.1073/pnas.79.6.2077openalex-snapshot
Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells.
inferredcites →https://doi.org/10.1073/pnas.81.20.6466openalex-snapshot
Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells.
inferredcites →https://doi.org/10.1073/pnas.87.6.2211openalex-snapshot
Site-specific integration by adeno-associated virus.
inferredcites →https://doi.org/10.1073/pnas.89.15.7257openalex-snapshot
Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector.
inferredcites →https://doi.org/10.1073/pnas.90.22.10613openalex-snapshot
Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector.
inferredcites →https://doi.org/10.1089/hum.1998.9.8-1181openalex-snapshot
Selective and Rapid Uptake of Adeno-Associated Virus Type 2 in Brain
inferredcites →https://doi.org/10.1089/hum.2010.245openalex-snapshot
Optimizing Promoters for Recombinant Adeno-Associated Virus-Mediated Gene Expression in the Peripheral and Central Nervous System Using Self-Complementary Vectors
inferredcites →https://doi.org/10.1089/hum.2012.107openalex-snapshot
Restoration of Cytoskeleton Homeostasis After Gigaxonin Gene Transfer for Giant Axonal Neuropathy
inferredcites →https://doi.org/10.1089/hum.2013.2515openalex-snapshot
An Emerging Adeno-Associated Viral Vector Pipeline for Cardiac Gene Therapy
inferredcites →https://doi.org/10.1089/hum.2014.047openalex-snapshot
The First Adeno-Associated Virus Gene Transfer Experiment, 1983
inferredcites →https://doi.org/10.1089/hum.2014.106openalex-snapshot
Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial
inferredcites →https://doi.org/10.1099/0022-1317-4-1-37openalex-snapshot
The DNA of a Minute Virus of Mice
inferredcites →https://doi.org/10.1111/j.1432-1033.1981.tb06443.xopenalex-snapshot
Adeno‐Associated Virus RNA Transcription in vivo
inferredcites →https://doi.org/10.1126/science.149.3685.754openalex-snapshot
Adenovirus-Associated Defective Virus Particles
inferredcites →https://doi.org/10.1126/scitranslmed.3003454openalex-snapshot
Long-Term Follow-Up After Gene Therapy for Canavan Disease
inferredcites →https://doi.org/10.1128/jvi.00675-12openalex-snapshot
Single Amino Acid Modification of Adeno-Associated Virus Capsid Changes Transduction and Humoral Immune Profiles
inferredcites →https://doi.org/10.1128/jvi.00687-10openalex-snapshot
Mutagenesis of Adeno-Associated Virus Type 2 Capsid Protein VP1 Uncovers New Roles for Basic Amino Acids in Trafficking and Cell-Specific Transduction
inferredcites →https://doi.org/10.1128/jvi.00843-06openalex-snapshot
Adeno-Associated Virus Type 2 Contains an Integrin α5β1 Binding Domain Essential for Viral Cell Entry
inferredcites →https://doi.org/10.1128/jvi.00935-12openalex-snapshot
Cytoplasmic Trafficking, Endosomal Escape, and Perinuclear Accumulation of Adeno-Associated Virus Type 2 Particles Are Facilitated by Microtubule Network
inferredcites →https://doi.org/10.1128/jvi.02309-08openalex-snapshot
Enhancement of Adeno-Associated Virus Infection by Mobilizing Capsids into and Out of the Nucleolus
inferredcites →https://doi.org/10.1128/jvi.02660-13openalex-snapshot
Recombinant Adeno-Associated Virus Utilizes Host Cell Nuclear Import Machinery To Enter the Nucleus
inferredcites →https://doi.org/10.1128/jvi.10.6.1118-1125.1972openalex-snapshot
Adenovirus-Associated Virus Multiplication IX. Extent of Transcription of the Viral Genome In Vivo
inferredcites →https://doi.org/10.1128/jvi.16.3.559-568.1975openalex-snapshot
Physical map and strand polarity of specific fragments of adenovirus-associated virus DNA produced by endonuclease R-EcoRI
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Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells
inferredcites →https://doi.org/10.1128/jvi.34.2.402-409.1980openalex-snapshot
Nucleotide sequence of the inverted terminal repetition in adeno-associated virus DNA
inferredcites →https://doi.org/10.1128/jvi.51.3.611-619.1984openalex-snapshot
Genetic analysis of adeno-associated virus: properties of deletion mutants constructed in vitro and evidence for an adeno-associated virus replication function
inferredcites →https://doi.org/10.1128/jvi.61.10.3096-3101.1987openalex-snapshot
A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication
inferredcites →https://doi.org/10.1128/jvi.61.8.2555-2558.1987openalex-snapshot
Identification of a repeated sequence element required for efficient encapsidation of the adenovirus type 5 chromosome
inferredcites →https://doi.org/10.1128/jvi.63.9.3822-3828.1989openalex-snapshot
Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression
inferredcites →https://doi.org/10.1128/jvi.70.11.8098-8108.1996openalex-snapshot
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
inferredcites →https://doi.org/10.1128/jvi.70.5.3227-3234.1996openalex-snapshot
Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors
inferredcites →https://doi.org/10.1128/jvi.71.2.941-948.1997openalex-snapshot
A novel 165-base-pair terminal repeat sequence is the sole cis requirement for the adeno-associated virus life cycle
inferredcites →https://doi.org/10.1128/jvi.71.7.5236-5243.1997openalex-snapshot
Role for highly regulated rep gene expression in adeno-associated virus vector production
inferredcites →https://doi.org/10.1128/jvi.72.2.1438-1445.1998openalex-snapshot
Membrane-Associated Heparan Sulfate Proteoglycan Is a Receptor for Adeno-Associated Virus Type 2 Virions
inferredcites →https://doi.org/10.1128/jvi.72.3.2224-2232.1998openalex-snapshot
Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus
inferredcites →https://doi.org/10.1128/jvi.74.18.8732-8739.2000openalex-snapshot
Novel Transcriptional Regulatory Signals in the Adeno-Associated Virus Terminal Repeat A/D Junction Element
inferredcites →https://doi.org/10.1128/jvi.74.6.2777-2785.2000openalex-snapshot
Infectious Entry Pathway of Adeno-Associated Virus and Adeno-Associated Virus Vectors
inferredcites →https://doi.org/10.1128/jvi.76.2.791-801.2002openalex-snapshot
Cross-Packaging of a Single Adeno-Associated Virus (AAV) Type 2 Vector Genome into Multiple AAV Serotypes Enables Transduction with Broad Specificity
inferredcites →https://doi.org/10.1128/jvi.77.1.423-432.2003openalex-snapshot
Marker Rescue of Adeno-Associated Virus (AAV) Capsid Mutants: a Novel Approach for Chimeric AAV Production
inferredcites →https://doi.org/10.1128/jvi.78.9.4421-4432.2004openalex-snapshot
Cross-Dressing the Virion: the Transcapsidation of Adeno-Associated Virus Serotypes Functionally Defines Subgroups
inferredcites →https://doi.org/10.1172/jci33138openalex-snapshot
Recombinant adeno-associated virus vectors induce functionally impaired transgene product–specific CD8+ T cells in mice
inferredcites →https://doi.org/10.1172/jci66611openalex-snapshot
Adeno-associated virus capsid antigen presentation is dependent on endosomal escape
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Single-copy transduction and expression of human gamma-globin in K562 erythroleukemia cells using recombinant adeno-associated virus vectors: the effect of mutations in NF-E2 and GATA-1 binding motifs within the hypersensitivity site 2 enhancer [published erratum appears in Blood 1995 Feb…
inferredcites →https://doi.org/10.1371/journal.pone.0007705openalex-snapshot
AAV Recombineering with Single Strand Oligonucleotides
inferredcites →https://doi.org/10.4049/jimmunol.0803965openalex-snapshot
Adeno-Associated Virus Capsid Structure Drives CD4-Dependent CD8+ T Cell Response to Vector Encoded Proteins
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Incoming claims: where this work appears in other papers’ reference lists and arguments.
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inferred← citeshttps://doi.org/10.1016/j.ekir.2024.04.024openalex-snapshot
Thrombotic Microangiopathy as an Emerging Complication of Viral Vector–Based Gene Therapy
inferred← citeshttps://doi.org/10.1016/j.gendis.2025.101612openalex-snapshot
In vivo production of CAR T cell: Opportunities and challenges
inferred← citeshttps://doi.org/10.1016/j.gene.2024.148865openalex-snapshot
Recent advances and applications of the CRISPR-Cas system in the gene therapy of blood disorders
inferred← citeshttps://doi.org/10.1016/j.heares.2024.109151openalex-snapshot
Gene therapy for hereditary hearing loss
inferred← citeshttps://doi.org/10.1016/j.hoc.2022.04.002openalex-snapshot
Adeno-Associated Virus Vector Design–Moving the Adeno-Associated Virus to a Bioengineered Therapeutic Nanoparticle
inferred← citeshttps://doi.org/10.1016/j.ijbiomac.2025.148329openalex-snapshot
Hydrophobically-modified hyaluronic acid as stabilizer for oil-in-water emulsions to improve epidermis biodistribution of actives
inferred← citeshttps://doi.org/10.1016/j.jaci.2016.03.053openalex-snapshot
Anti-hIgE gene therapy of peanut-induced anaphylaxis in a humanized murine model of peanut allergy
inferred← citeshttps://doi.org/10.1016/j.jconrel.2016.05.031openalex-snapshot
Barriers to inhaled gene therapy of obstructive lung diseases: A review
inferred← citeshttps://doi.org/10.1016/j.jconrel.2025.02.015openalex-snapshot
Engineering sialylated N-glycans on adeno-associated virus capsids for targeted gene delivery and therapeutic applications
inferred← citeshttps://doi.org/10.1016/j.lfs.2021.119142openalex-snapshot
Challenges in adeno-associated virus-based treatment of central nervous system diseases through systemic injection
inferred← citeshttps://doi.org/10.1016/j.mad.2021.111549openalex-snapshot
Applications of adeno-associated virus vector-mediated gene delivery for neurodegenerative diseases and psychiatric diseases: Progress, advances, and challenges
inferred← citeshttps://doi.org/10.1016/j.matdes.2021.109580openalex-snapshot
Ultrasound-triggered on-demand drug delivery using hydrogel microbeads with release enhancer
inferred← citeshttps://doi.org/10.1016/j.molcel.2016.07.004openalex-snapshot
Methods for Optimizing CRISPR-Cas9 Genome Editing Specificity
inferred← citeshttps://doi.org/10.1016/j.molmed.2020.09.010openalex-snapshot
Journey to the Center of the Cell: Tracing the Path of AAV Transduction
inferred← citeshttps://doi.org/10.1016/j.neulet.2021.135679openalex-snapshot
AAV8-mediated Atoh1 overexpression induces dose-dependent regeneration of vestibular hair cells in adult mice
inferred← citeshttps://doi.org/10.1016/j.neulet.2022.136746openalex-snapshot
Functional analysis of mutations endowing rAAV2-retro with retrograde tracing capacity
inferred← citeshttps://doi.org/10.1016/j.neuro.2024.02.001openalex-snapshot
Absence of functional deficits in rats following systemic administration of an AAV9 vector despite moderate peripheral nerve and dorsal root ganglia findings: A clinically silent peripheral neuropathy
inferred← citeshttps://doi.org/10.1016/j.neuron.2019.02.017openalex-snapshot
Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality
inferred← citeshttps://doi.org/10.1016/j.omtm.2017.07.003openalex-snapshot
Thermal Stability as a Determinant of AAV Serotype Identity
inferred← citeshttps://doi.org/10.1016/j.omtm.2017.09.006openalex-snapshot
Safety and Efficacy of AAV Retrograde Pancreatic Ductal Gene Delivery in Normal and Pancreatic Cancer Mice
inferred← citeshttps://doi.org/10.1016/j.omtm.2017.11.002openalex-snapshot
A Robust System for Production of Superabundant VP1 Recombinant AAV Vectors
inferred← citeshttps://doi.org/10.1016/j.omtm.2019.01.008openalex-snapshot
Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors
inferred← citeshttps://doi.org/10.1016/j.omtm.2020.05.004openalex-snapshot
Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2
inferred← citeshttps://doi.org/10.1016/j.omtm.2022.01.011openalex-snapshot
Systemic and local immune responses to intraocular AAV vector administration in non-human primates
inferred← citeshttps://doi.org/10.1016/j.omtm.2022.06.009openalex-snapshot
Emerging therapeutic potential of adeno-associated virus-mediated gene therapy in liver fibrosis
inferred← citeshttps://doi.org/10.1016/j.omtm.2023.07.007openalex-snapshot
Partial genome content within rAAVs impacts performance in a cell assay-dependent manner
inferred← citeshttps://doi.org/10.1016/j.omtm.2023.101129openalex-snapshot
Non-canonical amino acid incorporation into AAV5 capsid enhances lung transduction in mice
inferred← citeshttps://doi.org/10.1016/j.omtm.2024.101298openalex-snapshot
Evaluation of a rapid multi-attribute combinatorial high-throughput UV-Vis/DLS/SLS analytical platform for rAAV quantification and characterization
inferred← citeshttps://doi.org/10.1016/j.omtm.2025.101418openalex-snapshot
A novel platform for engineered AAV-based vaccines
inferred← citeshttps://doi.org/10.1016/j.omtm.2025.101419openalex-snapshot
Impact of DNase digestion on titer measurements of engineered adeno-associated virus serotypes
inferred← citeshttps://doi.org/10.1016/j.omtm.2025.101471openalex-snapshot
Characterization of factors that influence rAAV yield and quality when produced using rHSV co-infection
inferred← citeshttps://doi.org/10.1016/j.omtm.2025.101602openalex-snapshot
Impact of pre-existing immunity on safety and biodistribution of a single AAV9 vector intrathecal injection in cynomolgus monkeys
inferred← citeshttps://doi.org/10.1016/j.phrs.2024.107308openalex-snapshot
Brain gliomas: Diagnostic and therapeutic issues and the prospects of drug-targeted nano-delivery technology
inferred← citeshttps://doi.org/10.1016/j.preteyeres.2021.100975openalex-snapshot
Early and late stage gene therapy interventions for inherited retinal degenerations
inferred← citeshttps://doi.org/10.1016/j.preteyeres.2022.101136openalex-snapshot
Glaucomatous optic neuropathy: Mitochondrial dynamics, dysfunction and protection in retinal ganglion cells
inferred← citeshttps://doi.org/10.1016/j.rvsc.2020.12.026openalex-snapshot
Fusion expression of the two soluble viral receptors of porcine reproductive and respiratory syndrome virus with a single adeno-associated virus vector
inferred← citeshttps://doi.org/10.1016/j.snr.2024.100242openalex-snapshot
Single-capsid identification of full and empty status of recombinant adeno-associated viruses via resistive pulse sensing
inferred← citeshttps://doi.org/10.1016/j.ymthe.2017.02.005openalex-snapshot
Integration of a CD19 CAR into the TCR Alpha Chain Locus Streamlines Production of Allogeneic Gene-Edited CAR T Cells
inferred← citeshttps://doi.org/10.1016/j.ymthe.2017.03.012openalex-snapshot
In Vivo Excision of HIV-1 Provirus by saCas9 and Multiplex Single-Guide RNAs in Animal Models
inferred← citeshttps://doi.org/10.1016/j.ymthe.2019.09.016openalex-snapshot
Short-Term Local Expression of a PD-L1 Blocking Antibody from a Self-Replicating RNA Vector Induces Potent Antitumor Responses
inferred← citeshttps://doi.org/10.1016/j.ymthe.2022.09.015openalex-snapshot
AAV vectors: The Rubik’s cube of human gene therapy
inferred← citeshttps://doi.org/10.1016/j.ymthe.2023.12.015openalex-snapshot
Evolving membrane-associated accessory protein variants for improved adeno-associated virus production
inferred← citeshttps://doi.org/10.1016/j.ymthe.2024.05.032openalex-snapshot
Gene therapy for Lafora disease in the Epm2a mouse model
inferred← citeshttps://doi.org/10.1021/acs.analchem.2c04234openalex-snapshot
Analysis of AAV-Extracted DNA by Charge Detection Mass Spectrometry Reveals Genome Truncations
inferred← citeshttps://doi.org/10.1021/acs.analchem.3c05087openalex-snapshot
Multiple Ion Charge Extraction (MICE) for High-Throughput Charge Detection Mass Spectrometry
inferred← citeshttps://doi.org/10.1021/acs.analchem.6b00883openalex-snapshot
Resolving Adeno-Associated Viral Particle Diversity With Charge Detection Mass Spectrometry
inferred← citeshttps://doi.org/10.1021/acs.jpcb.6b10131openalex-snapshot
Probing the Link among Genomic Cargo, Contact Mechanics, and Nanoindentation in Recombinant Adeno-Associated Virus 2
inferred← citeshttps://doi.org/10.1021/acsnano.5b05558openalex-snapshot
Light-Activated Nuclear Translocation of Adeno-Associated Virus Nanoparticles Using Phytochrome B for Enhanced, Tunable, and Spatially Programmable Gene Delivery
inferred← citeshttps://doi.org/10.1021/acsnano.5c16533openalex-snapshot
Mechanisms of Adeno-Associated Virus Serotype 9 Vector Characterization and Quality Control through Solid-State Nanopores
inferred← citeshttps://doi.org/10.1038/mt.2015.179openalex-snapshot
Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids
inferred← citeshttps://doi.org/10.1038/mt.2015.182openalex-snapshot
Adeno-associated Vector Toxicity—To Be or Not to Be?
inferred← citeshttps://doi.org/10.1038/mt.2015.191openalex-snapshot
Host Anti-antibody Responses Following Adeno-associated Virus–mediated Delivery of Antibodies Against HIV and SIV in Rhesus Monkeys
inferred← citeshttps://doi.org/10.1038/mtm.2015.41openalex-snapshot
High-accuracy biodistribution analysis of adeno-associated virus variants by double barcode sequencing
inferred← citeshttps://doi.org/10.1038/mtna.2016.49openalex-snapshot
CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector
inferred← citeshttps://doi.org/10.1038/ng.3893openalex-snapshot
Identification of liver-specific enhancer–promoter activity in the 3′ untranslated region of the wild-type AAV2 genome
inferred← citeshttps://doi.org/10.1038/nn.4593openalex-snapshot
Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems
inferred← citeshttps://doi.org/10.1038/s41417-019-0108-8openalex-snapshot
AAV-mediated expression of 3TSR inhibits tumor and metastatic lesion development and extends survival in a murine model of epithelial ovarian carcinoma
inferred← citeshttps://doi.org/10.1038/s41434-020-0177-1openalex-snapshot
Gene therapy development in hearing research in China
inferred← citeshttps://doi.org/10.1038/s41434-021-00296-0openalex-snapshot
Rational engineering of a functional CpG-free ITR for AAV gene therapy
inferred← citeshttps://doi.org/10.1038/s41434-025-00519-8openalex-snapshot
A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice
inferred← citeshttps://doi.org/10.1038/s41467-024-48221-4openalex-snapshot
An adeno-associated virus variant enabling efficient ocular-directed gene delivery across species
inferred← citeshttps://doi.org/10.1038/s41573-019-0012-9openalex-snapshot
Adeno-associated virus vector as a platform for gene therapy delivery
inferred← citeshttps://doi.org/10.1038/s41576-019-0205-4openalex-snapshot
Engineering adeno-associated virus vectors for gene therapy
inferred← citeshttps://doi.org/10.1038/s41598-017-01004-yopenalex-snapshot
In vivo dynamics of AAV-mediated gene delivery to sensory neurons of the trigeminal ganglia
inferred← citeshttps://doi.org/10.1038/s41598-020-60721-zopenalex-snapshot
Distribution and genetic diversity of adeno-associated viruses in bats from coastal areas of Southeast China
inferred← citeshttps://doi.org/10.1038/s41598-020-70950-xopenalex-snapshot
Neurotoxicity of HIV-1 Tat is attributed to its penetrating property
inferred← citeshttps://doi.org/10.1038/s41598-020-78521-wopenalex-snapshot
A comparison of AAV-vector production methods for gene therapy and preclinical assessment
inferred← citeshttps://doi.org/10.1038/s41598-021-01220-7openalex-snapshot
Functional roles of the membrane-associated AAV protein MAAP
inferred← citeshttps://doi.org/10.1038/srep28272openalex-snapshot
Ultramicroscopy as a novel tool to unravel the tropism of AAV gene therapy vectors in the brain
inferred← citeshttps://doi.org/10.1039/d5pm00148jopenalex-snapshot
Brain-targeted intranasal delivery of biologics: a perspective for Alzheimer's disease treatment
inferred← citeshttps://doi.org/10.1073/pnas.1704766114openalex-snapshot
Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion
inferred← citeshttps://doi.org/10.1080/14737175.2020.1763794openalex-snapshot
Gene therapy for Parkinson’s disease: contemporary practice and emerging concepts
inferred← citeshttps://doi.org/10.1080/14737175.2023.2268276openalex-snapshot
Current and emerging targeted therapies for spinal muscular atrophy
inferred← citeshttps://doi.org/10.1089/biores.2020.0031openalex-snapshot
Two-Plasmid Packaging System for Recombinant Adeno-Associated Virus
inferred← citeshttps://doi.org/10.1089/hgtb.2016.173openalex-snapshot
A Scalable and Accurate Method for Quantifying Vector Genomes of Recombinant Adeno-Associated Viruses in Crude Lysate
inferred← citeshttps://doi.org/10.1089/hgtb.2016.188openalex-snapshot
Improving the Quality of Adeno-Associated Viral Vector Preparations: The Challenge of Product-Related Impurities
inferred← citeshttps://doi.org/10.1089/hgtb.2018.228openalex-snapshot
Standardized, Scalable, and Timely Flexible Adeno-Associated Virus Vector Production Using Frozen High-Density HEK-293 Cell Stocks and CELLdiscs
inferred← citeshttps://doi.org/10.1089/hgtb.2019.123openalex-snapshot
Development of Versatile and Flexible Sf9 Packaging Cell Line-Dependent OneBac System for Large-Scale Recombinant Adeno-Associated Virus Production
inferred← citeshttps://doi.org/10.1089/hum.2015.074openalex-snapshot
Delivery and Specificity of CRISPR/Cas9 Genome Editing Technologies for Human Gene Therapy
inferred← citeshttps://doi.org/10.1089/hum.2015.136openalex-snapshot
Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle
inferred← citeshttps://doi.org/10.1089/hum.2015.29014.kibopenalex-snapshot
Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?
inferred← citeshttps://doi.org/10.1089/hum.2016.107openalex-snapshot
Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles
inferred← citeshttps://doi.org/10.1089/hum.2017.29041.trfopenalex-snapshot
AAV Is Now a Medicine: We Had Better Get This Right
inferred← citeshttps://doi.org/10.1089/hum.2020.054openalex-snapshot
Detailed Protocol for the Novel and Scalable Viral Vector Upstream Process for AAV Gene Therapy Manufacturing
inferred← citeshttps://doi.org/10.1089/hum.2020.105openalex-snapshot
The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System
inferred← citeshttps://doi.org/10.1089/hum.2020.29109.intopenalex-snapshot
From the Cultural Revolution to the Gene Therapy Revolution: An Interview with Guangping Gao, PhD
inferred← citeshttps://doi.org/10.1089/hum.2021.169openalex-snapshot
Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors
inferred← citeshttps://doi.org/10.1089/hum.2021.219openalex-snapshot
Development of a Beta Cell-Specific Expression Control Element for Recombinant Adeno-Associated Virus
inferred← citeshttps://doi.org/10.1089/hum.2021.29147.hbuopenalex-snapshot
Gene and Cell Therapy for Inherited and Acquired Immune Deficiency
inferred← citeshttps://doi.org/10.1089/hum.2022.218openalex-snapshot
Application of Size Exclusion Chromatography with Multiangle Light Scattering in the Analytical Development of a Preclinical Stage Gene Therapy Program
inferred← citeshttps://doi.org/10.1089/hum.2022.223openalex-snapshot
Characteristics, Recombination Methods, and Applications Progresses of Split-Cas9 System
inferred← citeshttps://doi.org/10.1089/hum.2023.208openalex-snapshot
Efficacy and Safety of Adeno-Associated Virus-Based Clinical Gene Therapy for Hemophilia: A Systematic Review and Meta-Analysis
inferred← citeshttps://doi.org/10.1093/eurheartj/ehaf520openalex-snapshot
Pharmacotherapy of cardiovascular diseases from herbs and pills to nucleic acids
inferred← citeshttps://doi.org/10.1097/iio.0000000000000361openalex-snapshot
Viral Vector Technologies and Strategies: Improving on Nature
inferred← citeshttps://doi.org/10.1097/scs.0000000000006602openalex-snapshot
Rewriting the Future: Promises and Limits of Germline Gene Editing in Craniofacial Surgery
inferred← citeshttps://doi.org/10.1097/wnr.0000000000001359openalex-snapshot
Adeno-associated virus packaged TRPC5 gene therapy alleviated spinal cord ischemic reperfusion injury in rats
inferred← citeshttps://doi.org/10.1101/2022.08.27.505551openalex-snapshot
Spectral Interferences Impede the High-Resolution Mass Analysis of Recombinant Adeno-Associated Viruses
inferred← citeshttps://doi.org/10.1101/2023.12.14.571636openalex-snapshot
Gene replacement therapy for Lafora disease in the Epm2a -/- mouse model
inferred← citeshttps://doi.org/10.1101/2024.09.11.612416openalex-snapshot
The Role of Thermal Stability in AAV Titration of Engineered Variants
inferred← citeshttps://doi.org/10.1101/689653openalex-snapshot
Presynaptic mitochondria volume and abundance increase during development of a high-fidelity synapse
inferred← citeshttps://doi.org/10.1111/brv.12718openalex-snapshot
Humoral immune responses to AAV gene therapy in the ocular compartment
inferred← citeshttps://doi.org/10.1128/jvi.00943-19openalex-snapshot
A CRISPR Screen Identifies the Cell Polarity Determinant Crumbs 3 as an Adeno-associated Virus Restriction Factor in Hepatocytes
inferred← citeshttps://doi.org/10.1128/jvi.01568-16openalex-snapshot
Heparan Sulfate Binding Promotes Accumulation of Intravitreally Delivered Adeno-associated Viral Vectors at the Retina for Enhanced Transduction but Weakly Influences Tropism
inferred← citeshttps://doi.org/10.1146/annurev-neuro-080317-062048openalex-snapshot
Viral Strategies for Targeting the Central and Peripheral Nervous Systems
inferred← citeshttps://doi.org/10.1146/annurev-virology-101416-041929openalex-snapshot
Engineered Expression of Broadly Neutralizing Antibodies Against Human Immunodeficiency Virus
inferred← citeshttps://doi.org/10.1155/2024/5487973openalex-snapshot
Sustained and Efficient Delivery of Antivascular Endothelial Growth Factor by the Adeno‐associated Virus for the Treatment of Corneal Neovascularization: An Outlook for Its Clinical Translation
inferred← citeshttps://doi.org/10.11606/t.5.2019.tde-24062021-214254openalex-snapshot
Efeito da utilização de células-tronco derivadas de adipócitos no conduto de veia jugular glicerinada na regeneração do nervo ciático: estudo experimental
inferred← citeshttps://doi.org/10.1161/atvbaha.126.324442openalex-snapshot
Sustained Correction of Hereditary Antithrombin Deficiency in Mice by AAV8-Mediated Gene Delivery
inferred← citeshttps://doi.org/10.1164/rccm.201510-2024edopenalex-snapshot
Gene Therapy with Adeno-associated Virus for Cystic Fibrosis
inferred← citeshttps://doi.org/10.1176/appi.books.9781615375523.as01openalex-snapshot
Chapter 1. Basic Principles of Molecular Biology and Genomics
inferred← citeshttps://doi.org/10.1177/0192623318803867openalex-snapshot
Considerations for Preclinical Safety Assessment of Adeno-Associated Virus Gene Therapy Products
inferred← citeshttps://doi.org/10.1177/11206721211049001openalex-snapshot
The change of microglia numbers within the mice retina, optic nerve and chiasm following intravitreal AAV2-GFP injection
inferred← citeshttps://doi.org/10.1182/asheducation-2015.1.33openalex-snapshot
Emerging genetic and pharmacologic therapies for controlling hemostasis: beyond recombinant clotting factors
inferred← citeshttps://doi.org/10.1186/s12896-018-0479-1openalex-snapshot
Infectivity of adeno-associated virus serotypes in mouse testis
inferred← citeshttps://doi.org/10.1186/s12933-026-03130-2openalex-snapshot
Reactive carbonyl species in health and chronic disease: from methylglyoxal to an integrative network of metabolic regulation
inferred← citeshttps://doi.org/10.1186/s12935-021-01776-4openalex-snapshot
Adeno‐associated virus (AAV)-based gene therapy for glioblastoma
inferred← citeshttps://doi.org/10.1186/s12964-025-02246-4openalex-snapshot
Gene therapy for age-related macular degeneration: a promising frontier in vision preservation
inferred← citeshttps://doi.org/10.1186/s12985-021-01555-7openalex-snapshot
Optimization of AAV vectors to target persistent viral reservoirs
inferred← citeshttps://doi.org/10.1186/s12985-023-02196-8openalex-snapshot
A dual role for adeno-associated virus in human health
inferred← citeshttps://doi.org/10.1186/s13036-020-00245-2openalex-snapshot
Machine intelligence for nerve conduit design and production
inferred← citeshttps://doi.org/10.1186/s13395-015-0064-4openalex-snapshot
Differential myofiber-type transduction preference of adeno-associated virus serotypes 6 and 9
inferred← citeshttps://doi.org/10.1186/s42444-022-00060-4openalex-snapshot
Optogenetics in cardiology: methodology and future applications
inferred← citeshttps://doi.org/10.1186/s43141-023-00502-zopenalex-snapshot
A review of transgenic animal techniques and their applications
inferred← citeshttps://doi.org/10.1210/endrev/bnab030openalex-snapshot
Myostatin/Activin Receptor Ligands in Muscle and the Development Status of Attenuating Drugs
inferred← citeshttps://doi.org/10.1248/bpb.b18-00913openalex-snapshot
Adeno Associated Virus (AAV) as a Tool for Clinical and Experimental Delivery of Target Genes into the Mammalian Retina
inferred← citeshttps://doi.org/10.12750/jarb.38.1.2openalex-snapshot
Advances in research to restore vision
inferred← citeshttps://doi.org/10.1360/n052015-00091openalex-snapshot
The Application of Geminiviruses in Biotechnology
inferred← citeshttps://doi.org/10.1371/journal.ppat.1007988openalex-snapshot
Ring finger protein 121 is a potent regulator of adeno-associated viral genome transcription
inferred← citeshttps://doi.org/10.1517/21678707.2015.1039511openalex-snapshot
Recombinant adeno-associated virus vectors in the treatment of rare diseases
inferred← citeshttps://doi.org/10.1517/21678707.2015.1069179openalex-snapshot
Obstacles and future of gene therapy for hemophilia
inferred← citeshttps://doi.org/10.1523/jneurosci.0102-20.2020openalex-snapshot
Mouse γ-Synuclein Promoter-Mediated Gene Expression and Editing in Mammalian Retinal Ganglion Cells
inferred← citeshttps://doi.org/10.1523/jneurosci.0363-19.2019openalex-snapshot
Presynaptic Mitochondria Volume and Abundance Increase during Development of a High-Fidelity Synapse
inferred← citeshttps://doi.org/10.15581/10171/67875openalex-snapshot
Optimizing gene therapy for infant mice with citrullinemia type 1
inferred← citeshttps://doi.org/10.17615/cr64-be62openalex-snapshot
Biology of AAV Vectors in the Central Nervous System
inferred← citeshttps://doi.org/10.18231/j.ijrimcr.2022.024openalex-snapshot
Cost-Effective Approach to AAV Viral Vector Manufacturing
inferred← citeshttps://doi.org/10.21203/rs.3.rs-3697467/v1openalex-snapshot
NeuroD1 and Ascl1 Convert Human Glial Cells into Neurons in Ex Vivo Culture of Human Brain Tissue
inferred← citeshttps://doi.org/10.21248/gups.68021openalex-snapshot
Targeted local combination therapy with checkpoint inhibitors and CAR-NK cells in glioblastoma using DARPin-linked AAV vectors
inferred← citeshttps://doi.org/10.21248/gups.86442openalex-snapshot
Molecular concepts for pandemic viruses : membrane fusion assays and targeting of reservoir cells
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