Citation Impact
Citing Papers
SuFEx-enabled, agnostic discovery of covalent inhibitors of human neutrophil elastase
2019 StandoutNobel
Human Induced Pluripotent Stem Cells Free of Vector and Transgene Sequences
2009 StandoutScience
Diversity oriented clicking delivers β-substituted alkenyl sulfonyl fluorides as covalent human neutrophil elastase inhibitors
2022 StandoutNobel
Use of epitope libraries to identify exon-specific monoclonal antibodies for characterization of altered dystrophins in muscular dystrophy.
1993
Ultrastructural Characterization of the Lower Motor System in a Mouse Model of Krabbe Disease
2016 Standout
The RNA Binding Protein Quaking Regulates Formation of circRNAs
2015 Standout
Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: Implication for clinical trials
2010
Generation of Human Melanocytes from Induced Pluripotent Stem Cells
2011 StandoutNobel
A Novel Function for SMN, the Spinal Muscular Atrophy Disease Gene Product, in Pre-mRNA Splicing
1998
Mechanisms Underlying Inflammation in Neurodegeneration
2010 Standout
mRNA-based therapeutics — developing a new class of drugs
2014 StandoutNobel
Quantitative Analyses of SMN1 and SMN2 Based on Real-Time LightCycler PCR: Fast and Highly Reliable Carrier Testing and Prediction of Severity of Spinal Muscular Atrophy
2002 Standout
Modeling familial Alzheimer's disease with induced pluripotent stem cells
2011 StandoutNobel
Circular dystrophin RNAs consisting of exons that were skipped by alternative splicing
1999
Constructing and Deconstructing Stem Cell Models of Neurological Disease
2011
Gut microbiota metabolism of dietary fiber influences allergic airway disease and hematopoiesis
2014 Standout
Exclusion of Htra2-β1, an up-regulator of full-length SMN2 transcript, as a modifying gene for spinal muscular atrophy
2000
Alternative RNA splicing in the nervous system
2001
Detecting and characterizing circular RNAs
2014 Standout
The muscular dystrophies
2002 Standout
The Science of Stroke: Mechanisms in Search of Treatments
2010 Standout
Pharmacology of a Central Nervous System Delivered 2′-O-Methoxyethyl–Modified Survival of Motor Neuron Splicing Oligonucleotide in Mice and Nonhuman Primates
2014
Insights into the activation mechanism of class I HDAC complexes by inositol phosphates
2016
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
2011 Standout
Pyrrolidinyl‐Spirooxindole Natural Products as Inspirations for the Development of Potential Therapeutic Agents
2007 Standout
Neonatal screening for muscular dystrophy
1993
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
2006 StandoutNobel
Immunogenicity of induced pluripotent stem cells
2011 StandoutNature
An SMN-Dependent U12 Splicing Event Essential for Motor Circuit Function
2012
SMN Is Required for Sensory-Motor Circuit Function in Drosophila
2012
Rapid Single-Step Induction of Functional Neurons from Human Pluripotent Stem Cells
2013 StandoutNobel
A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2
1999
Ganglion-specific splicing of TRPV1 underlies infrared sensation in vampire bats
2011 StandoutNatureNobel
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
2016 Standout
Telomerase RNA structure and function: implications for dyskeratosis congenita
2004 StandoutNobel
The structural and functional diversity of dystrophin
1993
Anchor extension: a structure-guided approach to design cyclic peptides targeting enzyme active sites
2021 StandoutNobel
Targeted Exon Skipping in Transgenic hDMD Mice: A Model for Direct Preclinical Screening of Human-Specific Antisense Oligonucleotides
2004
Regulation of mRNA decapping
2010
Genotype–phenotype studies in infantile spinal muscular atrophy (SMA) type I in Germany: implications for clinical trials and genetic counselling
2009
The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn-/- mice and results in a mouse with spinal muscular atrophy
2000
In vitro pharmacologic testing using human induced pluripotent stem cell-derived cardiomyocytes
2009 StandoutNobel
Long-term persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation
2002
Sequence diversity in CYP3A promoters and characterization of the genetic basis of polymorphic CYP3A5 expression
2001 Standout
Multiple exon skipping and RNA circularisation contribute to the severe phenotypic expression of exon 5 dystrophin deletion
2003
The Regulation of Splice-Site Selection, and Its Role in Human Disease
1997
Induced pluripotent stem cells and reprogramming: seeing the science through the hype
2009 StandoutNobel
Hypoxia Rescues Frataxin Loss by Restoring Iron Sulfur Cluster Biogenesis
2019 StandoutNobel
Undetectable dystrophin can still result in a relatively benign phenotype of dystrophinopathy
1999
An ~140-kb deletion associated with feline spinal muscular atrophy implies an essentialLIX1function for motor neuron survival
2006
Induced pluripotent stem cells from a spinal muscular atrophy patient
2008 Nature
Spinal Muscular Atrophy: From Gene to Therapy
2006
An update of the mutation spectrum of the survival motor neuron gene (SMN1) in autosomal recessive spinal muscular atrophy (SMA)
2000
Genome-Scale Identification of SARS-CoV-2 and Pan-coronavirus Host Factor Networks
2020 StandoutNobel
A Fresh Look at iPS Cells
2009 StandoutNobel
Histone deacetylase inhibitors reverse gene silencing in Friedreich's ataxia
2006
The PtdIns(3,4)P2 phosphatase INPP4A is a suppressor of excitotoxic neuronal death
2010 StandoutNatureNobel
The Noncoding RNA Revolution—Trashing Old Rules to Forge New Ones
2014 Standout
Enhancement of SMN2 Exon 7 Inclusion by Antisense Oligonucleotides Targeting the Exon
2007
Correlation of SMNt and SMNc gene copy number with age of onset and survival in spinal muscular atrophy
1998
Alternative Splicing: Therapeutic Target and Tool
2006
MicroRNA maturation: stepwise processing and subcellular localization
2002 Standout
Cdk5 Regulates the Phosphorylation of Tyrosine 1472 NR2B and the Surface Expression of NMDA Receptors
2008
SMN Requirement for Synaptic Vesicle, Active Zone and Microtubule Postnatal Organization in Motor Nerve Terminals
2011
Parkinson's Disease Patient-Derived Induced Pluripotent Stem Cells Free of Viral Reprogramming Factors
2009 Standout
Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
2010
Induced pluripotent stem cells: a new revolution for clinical neurology?
2011
Using sulfuramidimidoyl fluorides that undergo sulfur(vi) fluoride exchange for inverse drug discovery
2020 StandoutNobel
Optically pure bulky (hetero)arylalkyl carbinols via kinetic resolution
2011
Hepatic Stellate Cells: Protean, Multifunctional, and Enigmatic Cells of the Liver
2008 Standout
TMEM41B Is a Pan-flavivirus Host Factor
2020 StandoutNobel
The evolution of gene expression levels in mammalian organs
2011 StandoutNatureNobel
The survival of motor neurons (SMN) protein interacts with the snoRNP proteins fibrillarin and GAR1
2001
Comparative Analysis of Antisense Oligonucleotide Sequences for Targeted Skipping of Exon 51 During Dystrophin Pre-mRNA Splicing in Human Muscle
2007
Iron Catalysis in Organic Synthesis
2015 Standout
A mouse model for spinal muscular atrophy
2000
Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy
2011
Chemical Genetics
2006
Long-term persistence of donor nuclei in a Duchenne muscular dystrophy patient receiving bone marrow transplantation
2002
Massive Idiosyncratic Exon Skipping Corrects the Nonsense Mutation in Dystrophic Mouse Muscle and Produces Functional Revertant Fibers by Clonal Expansion
2000
A stoichiometric complex of neurexins and dystroglycan in brain
2001 StandoutNobel
Recent Stem Cell Advances: Induced Pluripotent Stem Cells for Disease Modeling and Stem Cell–Based Regeneration
2010 StandoutNobel
A Functional Interaction between the Survival Motor Neuron Complex and RNA Polymerase II
2001
Function and Genetics of Dystrophin and Dystrophin-Related Proteins in Muscle
2002
A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy
1999
Identification of simple arylfluorosulfates as potent agents against resistant bacteria
2021 StandoutNobel
Nuclear repartitioning of galectin-1 by an extracellular glycan switch regulates mammary morphogenesis
2016 StandoutNobel
A Heck–Matsuda Process for the Synthesis of β‐Arylethenesulfonyl Fluorides: Selectively Addressable Bis‐electrophiles for SuFEx Click Chemistry
2016 StandoutNobel
Impact of Natural Products on Developing New Anti-Cancer Agents
2009 Standout
Short-chain fatty acids enhance nuclear receptor activity through mitogen-activated protein kinase activation and histone deacetylase inhibition
2004
Neurons generated by direct conversion of fibroblasts reproduce synaptic phenotype caused by autism-associated neuroligin-3 mutation
2013 StandoutNobel
Cellular and Molecular Regulation of Muscle Regeneration
2004 Standout
Arylfluorosulfates Inactivate Intracellular Lipid Binding Protein(s) through Chemoselective SuFEx Reaction with a Binding Site Tyr Residue
2016 StandoutNobel
Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse
2000
Htra2-β 1 stimulates an exonic splicing enhancer and can restore full-length SMN expression to survival motor neuron 2 ( SMN2 )
2000
When Is a Deletion Not a Deletion? When It Is Converted
1997
Mechanisms of Alternative Pre-Messenger RNA Splicing
2003 Standout
Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles
2004 StandoutNobel
Rational Targeting of Active-Site Tyrosine Residues Using Sulfonyl Fluoride Probes
2015
A New Portal to SuFEx Click Chemistry: A Stable Fluorosulfuryl Imidazolium Salt Emerging as an “F−SO2+” Donor of Unprecedented Reactivity, Selectivity, and Scope
2017 StandoutNobel
Treatment of spinal muscular atrophy by sodium butyrate
2001
Works of Daniel D. Coovert being referenced
Diverse small-molecule modulators of SMN expression found by high-throughput compound screening: early leads towards a therapeutic for spinal muscular atrophy
2005
Characterization of translational frame exception patients in Duchenne/Becker muscular dystrophy
1993
Dystrophin expression and somatic reversion in prednisone‐treated and untreated Duchenne dystrophy
1991
SMNΔ7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN
2005
The survival motor neuron protein in spinal muscular atrophy
1997
Indoprofen Upregulates the Survival Motor Neuron Protein through a Cyclooxygenase-Independent Mechanism
2004
Valproic acid increases SMN levels in spinal muscular atrophy patient cells
2003
Synthesis and Biological Evaluation of Novel 2,4-Diaminoquinazoline Derivatives as SMN2 Promoter Activators for the Potential Treatment of Spinal Muscular Atrophy
2008
Somatic reversion/suppression in Duchenne muscular dystrophy (DMD): evidence supporting a frame-restoring mechanism in rare dystrophin-positive fibers.
1992