Citation Impact
Citing Papers
Systemic delivery of factor IX messenger RNA for protein replacement therapy
2017
Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons
2009
Murine liver repair via transient activation of regenerative pathways in hepatocytes using lipid nanoparticle-complexed nucleoside-modified mRNA
2021 StandoutNobel
Nucleoside-modified VEGFC mRNA induces organ-specific lymphatic growth and reverses experimental lymphedema
2021 StandoutNobel
Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity
2017
Oligonucleotide conjugated multi-functional adeno-associated viruses
2018
Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras
2018
Recurrent inhibitory circuitry as a mechanism for grid formation
2013 StandoutNobel
Lymphatic vasculature mediates macrophage reverse cholesterol transport in mice
2013 Standout
Development and Optimization of a Real-Time Quantitative PCR-Based Method for the Titration of AAV-2 Vector Stocks
2002
MYC on the Path to Cancer
2012 Standout
Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver
2010
Molecular imaging in living subjects: seeing fundamental biological processes in a new light
2003 Standout
Adjuvant-carrying synthetic vaccine particles augment the immune response to encapsulated antigen and exhibit strong local immune activation without inducing systemic cytokine release
2014
DNA-based asymmetric catalysis
2010 StandoutNobel
Pathogenesis of persistent lymphatic vessel hyperplasia in chronic airway inflammation
2005 Standout
Inflammation and Cancer: Triggers, Mechanisms, and Consequences
2019 Standout
mRNA vaccines — a new era in vaccinology
2018 StandoutNobel
Gene therapy for neurological disorders: progress and prospects
2018
Cancer nanomedicine: progress, challenges and opportunities
2016 Standout
Therapeutic microRNA Delivery Suppresses Tumorigenesis in a Murine Liver Cancer Model
2009
Wound healing and its impairment in the diabetic foot
2005 Standout
Computational Models of Grid Cells
2011 StandoutNobel
Adjuvant lipidoid-substituted lipid nanoparticles augment the immunogenicity of SARS-CoV-2 mRNA vaccines
2023 StandoutNobel
Lipid nanoparticle chemistry determines how nucleoside base modifications alter mRNA delivery
2021 StandoutNobel
Inter-Society Consensus for the Management of Peripheral Arterial Disease (TASC II)
2007 Standout
Principles of nanoparticle design for overcoming biological barriers to drug delivery
2015 Standout
Synaptotagmin Has an Essential Function in Synaptic Vesicle Positioning for Synchronous Release in Addition to Its Role as a Calcium Sensor
2009 Nobel
Translational Potential of Immune Tolerance Induction by AAV Liver-Directed Factor VIII Gene Therapy for Hemophilia A
2020
Long-Term Rescue of a Lethal Inherited Disease by Adeno-Associated Virus–Mediated Gene Transfer in a Mouse Model of Molybdenum-Cofactor Deficiency
2007
VEGF: necessary to prevent motoneuron degeneration, sufficient to treat ALS?
2004
DNA-based therapeutics and DNA delivery systems: A comprehensive review
2005
Single-spike detection in vitro and in vivo with a genetic Ca2+ sensor
2008 StandoutNobel
Virus-mediated gene delivery for human gene therapy
2012
An ionizable lipid toolbox for RNA delivery
2021 StandoutNobel
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
2008
Amniotic fluid stabilized lipid nanoparticles for in utero intra-amniotic mRNA delivery
2021 StandoutNobel
mRNA vaccines for infectious diseases: principles, delivery and clinical translation
2021 StandoutNobel
Lymphangiogenesis in development and human disease
2005 StandoutNature
In vivo reprogramming of adult pancreatic exocrine cells to β-cells
2008 StandoutNature
Induction of uncoupling protein 1 by central interleukin-6 gene delivery is dependent on sympathetic innervation of brown adipose tissue and underlies one mechanism of body weight reduction in rats
2002
A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
2016
Promise and problems associated with the use of recombinant AAV for the delivery of anti-HIV antibodies
2016
Angiogenesis in life, disease and medicine
2005 StandoutNature
Neocortical excitation/inhibition balance in information processing and social dysfunction
2011 StandoutNature
Vector-mediated gene transfer engenders long-lived neutralizing activity and protection against SIV infection in monkeys
2009
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
2003
Vector Design Tour de Force: Integrating Combinatorial and Rational Approaches to Derive Novel Adeno-associated Virus Variants
2014
In vivo genome editing using Staphylococcus aureus Cas9
2015 StandoutNature
Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
2018
Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
2013
VEGF-A stimulates lymphangiogenesis and hemangiogenesis in inflammatory neovascularization via macrophage recruitment
2004 Standout
The potential of adeno-associated viral vectors for gene delivery to muscle tissue
2014
Genome engineering using the CRISPR-Cas9 system
2013 Standout
Applications of viral nanoparticles in medicine
2011
MicroRNA therapeutics: towards a new era for the management of cancer and other diseases
2017 Standout
Non-coding RNAs in human disease
2011 Standout
Mitochondrial Membrane Permeabilization in Cell Death
2007 Standout
Correction of the Enzymatic and Functional Deficits in a Model of Pompe Disease Using Adeno-associated Virus Vectors
2002
AAV-Delivered Antibody Mediates Significant Protective Effects against SIVmac239 Challenge in the Absence of Neutralizing Activity
2015
Gene transfer as a tool to induce therapeutic vascular growth
2003
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
2011
Adeno-associated virus vector as a platform for gene therapy delivery
2019 Standout
microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors
2010
Induced pluripotent stem cell–derived hepatocytes have the functional and proliferative capabilities needed for liver regeneration in mice
2010 StandoutNobel
A Hybrid Vector System Expands Adeno-associated Viral Vector Packaging Capacity in a Transgene-independent Manner
2007
Effect of Genome Size on AAV Vector Packaging
2009
CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes
2016 Standout
Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9
2015
Gold Nanoparticles for Biology and Medicine
2010 Standout
Promoterless gene targeting without nucleases ameliorates haemophilia B in mice
2014 Nature
Probing the Functional Equivalence of Otoferlin and Synaptotagmin 1 in Exocytosis
2011 StandoutNobel
Targeted Retrograde Gene Delivery for Neuronal Protection
2002
Emerging Issues in AAV-Mediated In Vivo Gene Therapy
2017
Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics
2001
Optogenetics in Neural Systems
2011 Standout
Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system
2016
Single-polarity Recombinant Adeno-associated Virus 2 Vector-mediated Transgene Expression In Vitro and In Vivo: Mechanism of Transduction
2007
Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity
2017
Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial
2017
Optimizing Promoters for Recombinant Adeno-Associated Virus-Mediated Gene Expression in the Peripheral and Central Nervous System Using Self-Complementary Vectors
2011
Adeno-Associated Virus 2-Mediated Intratumoral Prostate Cancer Gene Therapy: Long-Term Maspin Expression Efficiently Suppresses Tumor Growth
2005
Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success—A Personal Perspective
2015
Comparison of Adenoviral and Adeno-Associated Viral Vectors for Pancreatic Gene Delivery In Vivo
2004
A model for gene therapy of human hereditary lymphedema
2001
Optogenetic Dissection of Entorhinal-Hippocampal Functional Connectivity
2013 StandoutScienceNobel
Ionizable Lipid Nanoparticles for In Vivo mRNA Delivery to the Placenta during Pregnancy
2023 StandoutNobel
Erratum: Gene therapy for neurological disorders: progress and prospects
2018
Adenoviral VEGF‐C overexpression induces blood vessel enlargement, tortuosity, and leakiness but no sprouting angiogenesis in the skin or mucous membranes
2002
Infectious Molecular Clones of Adeno-Associated Virus Isolated Directly from Human Tissues
2008
Involvement of Cellular Double-Stranded DNA Break Binding Proteins in Processing of the Recombinant Adeno-Associated Virus Genome
2001
The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9–dependent innate immune responses in the liver
2011
Gene transfer in tissue repair: status, challenges and future directions
2004
Degradable Controlled-Release Polymers and Polymeric Nanoparticles: Mechanisms of Controlling Drug Release
2016 Standout
Intratumoral activation of the necroptotic pathway components RIPK1 and RIPK3 potentiates antitumor immunity
2019 StandoutNobel
Brown Adipose Tissue: Function and Physiological Significance
2004 Standout
Recent Stem Cell Advances: Induced Pluripotent Stem Cells for Disease Modeling and Stem Cell–Based Regeneration
2010 StandoutNobel
Adeno-Associated Virus Structural Biology as a Tool in Vector Development
2013
Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
2013
In Vivo Potency Assay for Adeno-Associated Virus–Based Gene Therapy Vectors Using AAVrh.10 as an Example
2018
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
2002
Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template
2015
Lymphangiogenic Gene Therapy With Minimal Blood Vascular Side Effects
2002
Carbon Nanomaterials for Biological Imaging and Nanomedicinal Therapy
2015 Standout
Viral Vectors for Gene Therapy: Translational and Clinical Outlook
2015
Endothelial Hypoxia-Inducible Factor-2α Is Required for the Maintenance of Airway Microvasculature
2019 StandoutNobel
Activatable Photosensitizers for Imaging and Therapy
2010 Standout
Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver
2005
Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery
2017 StandoutNobel
Oligonucleotide-Modified Gold Nanoparticles for Intracellular Gene Regulation
2006 StandoutScience
Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing
2020
An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo
2015
Resolving Adeno-Associated Viral Particle Diversity With Charge Detection Mass Spectrometry
2016
Phenotypic correction of murine hemophilia A using an iPS cell-based therapy
2009
Role of the Vascular Endothelial Growth Factor Pathway in Tumor Growth and Angiogenesis
2004 Standout
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
2003
Structure-guided SCHEMA recombination generates diverse chimeric channelrhodopsins
2017 StandoutNobel
Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape
2017
High Prevalence of Infectious Adeno-associated Virus (AAV) in Human Peripheral Blood Mononuclear Cells Indicative of T Lymphocytes as Sites of AAV Persistence
2016
Works of Paul E. Monahan being referenced
Men with severe hemophilia in the United States: birth cohort analysis of a large national database
2016
Animal Models of Hemophilia
2011
Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia
2011
Persistent expression of canine factor IX in hemophilia B canines
1999
Adeno-associated virus vectors for gene therapy: more pros than cons?
2000
Proteasome Inhibitors Enhance Gene Delivery by AAV Virus Vectors Expressing Large Genomes in Hemophilia Mouse and Dog Models: A Strategy for Broad Clinical Application
2010
AAV vectors: is clinical success on the horizon?
2000
Transgene expression levels and kinetics determine risk of humoral immune response modeled in factor IX knockout and missense mutant mice
2006
Hemophilia gene therapy: Update
2002
Optimization of Self-complementary AAV Vectors for Liver-directed Expression Results in Sustained Correction of Hemophilia B at Low Vector Dose
2007
Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
2011
Abnormal hemostasis in a knock‐in mouse carrying a variant of factor IX with impaired binding to collagen type IV
2009
Sustained and Complete Phenotype Correction of Hemophilia B Mice Following Intramuscular Injection of AAV1 Serotype Vectors
2001
Gene therapy in an era of emerging treatment options for hemophilia B
2015
Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
2003
Evaluation of Risks Related to the Use of Adeno-Associated Virus-Based Vectors
2003
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
2014
Cellular immune response to cryptic epitopes during therapeutic gene transfer
2009
Kinetics of Recombinant Adeno-Associated Virus-Mediated Gene Transfer
2000
Single Amino Acid Modification of Adeno-Associated Virus Capsid Changes Transduction and Humoral Immune Profiles
2012