Citation Impact
Citing Papers
Viral Vectors in Gene Therapy: Where Do We Stand in 2023?
2023
Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System
2011
Telomerase gene therapy in adult and old mice delays aging and increases longevity without increasing cancer
2012
Screening Libraries to Discover Molecular Design Principles for the Targeted Delivery of mRNA with One-Component Ionizable Amphiphilic Janus Dendrimers Derived from Plant Phenolic Acids
2023 StandoutNobel
Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid
2016 StandoutNobel
Mechanisms, regulation and functions of the unfolded protein response
2020 Standout
In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy
2016
Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS
2013
AAV9-mediated telomerase activation does not accelerate tumorigenesis in the context of oncogenic K-Ras-induced lung cancer
2018
Gene therapy for neurological disorders: progress and prospects
2018
Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain
2016
Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery
2011
Signaling mechanisms controlling cranial placode neurogenesis and delamination
2013
Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB)
2009
Hallmarks of aging: An expanding universe
2023 Standout
Applications of CRISPR-Cas Enzymes in Cancer Therapeutics and Detection
2018 StandoutNobel
Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain
2017
Cellular senescence in ageing: from mechanisms to therapeutic opportunities
2020 Standout
Gene Therapy Strategies to Restore ER Proteostasis in Disease
2018
Evolution of a designed protein assembly encapsulating its own RNA genome
2017 StandoutNatureNobel
Alzheimer Disease: An Update on Pathobiology and Treatment Strategies
2019 Standout
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
2016 Standout
Non-viral vectors for gene-based therapy
2014 Standout
A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates
2018
Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan
2015
Perinatal systemic gene delivery using adeno-associated viral vectors
2014
In vivo genome editing using Staphylococcus aureus Cas9
2015 StandoutNature
Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
2013
Genome engineering using the CRISPR-Cas9 system
2013 Standout
Myasthenia gravis
2019 Standout
The AAV Vector Toolkit: Poised at the Clinical Crossroads
2012
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
MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression
2010 Nobel
Structures and mechanisms of glycosyl hydrolases
1995 Standout
The Hallmarks of Aging
2013 Standout
LYSOSOMAL STORAGE DISEASES
1991
Optimizing Promoters for Recombinant Adeno-Associated Virus-Mediated Gene Expression in the Peripheral and Central Nervous System Using Self-Complementary Vectors
2011
Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease
2015
Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain
2017
Adeno-Associated Virus Gene Therapy for Liver Disease
2016
Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates
2011
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
2016
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
2018
The Fibroblast Growth Factor signaling pathway
2015 Standout
Estrogen-mediated downregulation of AIRE influences sexual dimorphism in autoimmune diseases
2016
Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
2011
Erratum: Gene therapy for neurological disorders: progress and prospects
2018
AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease
2018
State-of-the-art human gene therapy: part I. Gene delivery technologies.
2015
Lectins: Carbohydrate-Specific Proteins That Mediate Cellular Recognition
1998 Standout
A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB
2015
Ionizable lipid nanoparticles deliver mRNA to pancreatic β cells via macrophage-mediated gene transfer
2023 StandoutNobel
In vivo genome editing improves motor function and extends survival in a mouse model of ALS
2017
Ionizable lipid nanoparticles for in utero mRNA delivery
2021 StandoutNobel
Adverse Health Consequences of Performance-Enhancing Drugs: An Endocrine Society Scientific Statement
2013 Standout
AAV9 delivering a modified human Mullerian inhibiting substance as a gene therapy in patient-derived xenografts of ovarian cancer
2015
Spinal muscular atrophy: Diagnosis and management in a new therapeutic era
2014
CRISPR-Cas guides the future of genetic engineering
2018 StandoutScienceNobel
Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape
2017
Works of Sandra Duqué being referenced
Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons
2009
Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders
2011
Intramuscular scAAV9-SMN Injection Mediates Widespread Gene Delivery to the Spinal Cord and Decreases Disease Severity in SMA Mice
2013
A Single Intravenous AAV9 Injection Mediates Bilateral Gene Transfer to the Adult Mouse Retina
2013
Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice
2010
A large animal model of spinal muscular atrophy and correction of phenotype
2014
Delamination of cells from neurogenic placodes does not involve an epithelial-to-mesenchymal transition
2007
Electrophysiological biomarkers in spinal muscular atrophy: proof of concept
2013