Citation Impact
Citing Papers
Systemic delivery of factor IX messenger RNA for protein replacement therapy
2017
Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion
2017
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
2015 Science
ASL mRNA-LNP Therapeutic for the Treatment of Argininosuccinic Aciduria Enables Survival Benefit in a Mouse Model
2023 StandoutNobel
Bone grafts and biomaterials substitutes for bone defect repair: A review
2017 Standout
Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients
2013
Oligonucleotide conjugated multi-functional adeno-associated viruses
2018
Myocardial Delivery of Lipidoid Nanoparticle Carrying modRNA Induces Rapid and Transient Expression
2015
Switchable Cas9
2017 StandoutNobel
Bone regeneration: current concepts and future directions
2011 Standout
The promise and challenge of therapeutic genome editing
2020 StandoutNatureNobel
Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery
2013
Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9
2014 Nobel
Recent Advances in Gene Delivery for Structural Bone Allografts
2007
Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid
2016 StandoutNobel
A guide to genome engineering with programmable nucleases
2014 Standout
A multifunctional AAV–CRISPR–Cas9 and its host response
2016
Inflammation and Cancer: Triggers, Mechanisms, and Consequences
2019 Standout
mRNA vaccines — a new era in vaccinology
2018 StandoutNobel
Adeno-Associated Virus Gene Therapy: Translational Progress and Future Prospects in the Treatment of Heart Failure
2018
Lipid nanoparticles for mRNA delivery
2021 Standout
Lipid nanoparticle chemistry determines how nucleoside base modifications alter mRNA delivery
2021 StandoutNobel
Self-deploying shape memory polymer scaffolds for grafting and stabilizing complex bone defects: A mouse femoral segmental defect study
2015
Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
2020 Standout
Applications of CRISPR-Cas Enzymes in Cancer Therapeutics and Detection
2018 StandoutNobel
Bone morphogenetic proteins and tissue engineering: future directions
2009
Highly efficient CD4+ T cell targeting and genetic recombination using engineered CD4+ cell-homing mRNA-LNPs
2021 StandoutNobel
Renal impairment, worsening renal function, and outcome in patients with heart failure: an updated meta-analysis
2013 Standout
Large-scale recombinant adeno-associated virus production
2011
mRNA vaccines for infectious diseases: principles, delivery and clinical translation
2021 StandoutNobel
Non-viral vectors for gene-based therapy
2014 Standout
Gene Therapy With Regulatory T Cells: A Beneficial Alliance
2018
Identification of preexisting adaptive immunity to Cas9 proteins in humans
2019
Promise and problems associated with the use of recombinant AAV for the delivery of anti-HIV antibodies
2016
Human Immune Responses to Adeno-Associated Virus (AAV) Vectors
2020
Targeting apoptosis in cancer therapy
2020 Standout
The effect of mesenchymal stem cells delivered via hydrogel-based tissue engineered periosteum on bone allograft healing
2013
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
Gene therapy for hemophilia
2015
Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
2013
The effect of mesenchymal stem cell sheets on structural allograft healing of critical sized femoral defects in mice
2014
CpG-depleted adeno-associated virus vectors evade immune detection
2013
The potential of adeno-associated viral vectors for gene delivery to muscle tissue
2014
Gene Therapy Leaves a Vicious Cycle
2019
The AAV Vector Toolkit: Poised at the Clinical Crossroads
2012
AAV-Delivered Antibody Mediates Significant Protective Effects against SIVmac239 Challenge in the Absence of Neutralizing Activity
2015
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
The AAV-mediated and RNA-guided CRISPR/Cas9 system for gene therapy of DMD and BMD
2017
Applications of CRISPR technologies in research and beyond
2016 StandoutNobel
Induction of Immune Tolerance to Foreign Protein via Adeno-Associated Viral Vector Gene Transfer in Mid-Gestation Fetal Sheep
2017
Variability in Cardiac miRNA-122 Level Determines Therapeutic Potential of miRNA-Regulated AAV Vectors
2020
CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes
2016 Standout
Cornerstones of CRISPR–Cas in drug discovery and therapy
2016 StandoutNobel
Therapeutic developments for Duchenne muscular dystrophy
2019
Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes
2017 StandoutNobel
Emerging Issues in AAV-Mediated In Vivo Gene Therapy
2017
The Emerging Role of In Vitro-Transcribed mRNA in Adoptive T Cell Immunotherapy
2019 StandoutNobel
Targeted gene therapies: tools, applications, optimization
2012
Early drop in systolic blood pressure and worsening renal function in acute heart failure: renal results of Pre‐RELAX‐AHF
2011
Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial
2017
Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development
2021 StandoutNobel
Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success—A Personal Perspective
2015
Electrospinning and Electrospun Nanofibers: Methods, Materials, and Applications
2019 Standout
Ionizable lipid nanoparticles deliver mRNA to pancreatic β cells via macrophage-mediated gene transfer
2023 StandoutNobel
Intratumoral activation of the necroptotic pathway components RIPK1 and RIPK3 potentiates antitumor immunity
2019 StandoutNobel
Adeno-Associated Virus Structural Biology as a Tool in Vector Development
2013
Ionizable lipid nanoparticles for in utero mRNA delivery
2021 StandoutNobel
Adverse Health Consequences of Performance-Enhancing Drugs: An Endocrine Society Scientific Statement
2013 Standout
Immune responses to AAV vectors: overcoming barriers to successful gene therapy
2013
Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template
2015
Viral Vectors for Gene Therapy: Translational and Clinical Outlook
2015
Guidelines for assessment of bone microstructure in rodents using micro–computed tomography
2010 Standout
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models
2015
Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery
2017 StandoutNobel
Mir‐142‐3p target sequences reduce transgene‐directed immunogenicity following intramuscular adeno‐associated virus 1 vector‐mediated gene delivery
2013
Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape
2017
Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery
2014 StandoutNobel
Works of Jade Samulski being referenced
Cardiac I-1c Overexpression With Reengineered AAV Improves Cardiac Function in Swine Ischemic Heart Failure
2014
The effect of surface demineralization of cortical bone allograft on the properties of recombinant adeno-associated virus coatings
2008
Dystrophin Immunity in Duchenne's Muscular Dystrophy
2010
Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
2011
Prediction of adeno-associated virus neutralizing antibody activity for clinical application
2015
Self-complementary AAV2.5-BMP2-coated Femoral Allografts Mediated Superior Bone Healing Versus Live Autografts in Mice With Equivalent Biomechanics to Unfractured Femur
2011
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